CELL SEARCH SYSTEM -CELL BANK- (RIKEN BRC) [RCB4557 : HUDEP-2]

21539 Deborah E Daniels, Damien J Downes, Ivan Ferrer-Vicens, Daniel C J Ferguson, Belinda K Singleton, Marieangela C Wilson, Kongtana Trakarnsanga, Ryo Kurita, Yukio Nakamura, David J Anstee, Jan Frayne Erratum to: Comparing the two leading erythroid lines BEL-A and HUDEP-2 Haematologica 2024 Jan 1;109(1):364 PubMed ID: 38186343 DOI: 10.3324/haematol.2023.284510

21906 Thijs C. J. Verheul, Nynke Gillemans, Kerstin Putzker, Rezin Majied, Tingyue Li, Memnia Vasiliou, Bert Eussen, Annelies de Klein, Wilfred F. J. van IJcken, Emile van den Akker, Marieke von Lindern, Joe Lewis, Ulrike Uhrig, Yukio Nakamura, Thamar van Dijk, Sjaak Philipsen A cellular reporter system to evaluate endogenous fetal hemoglobin induction and screen for therapeutic compounds Hemasphere 2024 Aug 6;8(8):e139 PubMed ID: 39108322 DOI: 10.1002/hem3.139

18128 Papaioannou NY, Patsali P, Naiisseh B, Papasavva PL, Koniali L, Kurita R, Nakamura Y, Christou S, Sitarou M, Mussolino C, Cathomen T, Kleanthous M, Lederer CW. High-efficiency editing in hematopoietic stem cells and the HUDEP-2 cell line based on in vitro mRNA synthesis Front Genome Ed 2023 5:1141618 PubMed ID: 36969374 DOI: 10.3389/fgeed.2023.1141618

18359 Venkatesan V, Christopher AC, Rhiel M, Azhagiri MKK, Babu P, Walavalkar K, Saravanan B, Andrieux G, Rangaraj S, Srinivasan S, Karuppusamy KV, Jacob A, Bagchi A, Pai AA, Nakamura Y, Kurita R, Balasubramanian P, Pai R, Marepally SK, Mohankumar KM, Velayudhan SR, Boerries M, Notani D, Cathomen T, Srivastava A, Thangavel S. Editing the core region in HPFH deletions alters fetal and adult globin expression for treatment of β-hemoglobinopathies Mol Ther Nucleic Acids 2023 32:671-688 PubMed ID: 37215154 DOI: 10.1016/j.omtn.2023.04.024

18360 Lv X, Murphy K, Murphy Z, Getman MR, Rahman NF, Nakamura Y, Blanc L, Gallagher PG, Palis J, Mohandas N, Steiner LA. HEXIM1 is an Essential Transcription Regulator During Human Erythropoiesis Blood 2023 blood.2022019495 PubMed ID: 37738561 DOI: 10.1182/blood.2022019495

18441 Zhang X, Xia F, Zhang X, Blumenthal RM, Cheng X. C2H2 zinc finger transcription factors associated with hemoglobinopathies J Mol Biol 2023 168343 PubMed ID: 37924864 DOI: 10.1016/j.jmb.2023.168343

18495 Lu HY, Orkin SH, Sankaran VG. Fetal Hemoglobin Regulation in Beta-Thalassemia Hematol Oncol Clin North Am 2023 37(2):301-312 PubMed ID: 36907604 DOI: 10.1016/j.hoc.2022.12.002

18497 Zeng S, Lei S, Qu C, Wang Y, Teng S, Huang P. CRISPR/Cas-based gene editing in therapeutic strategies for beta-thalassemia Hum Genet 2023 Oct 25 PubMed ID: 37878144 DOI: 10.1007/s00439-023-02610-9

18508 Arif T, Farooq A, Ahmad FJ, Akhtar M, Choudhery MS. Prime editing: A potential treatment option for β-thalassemia Cell Biol Int 2023 47(4):699-713 PubMed ID: 36480796 DOI: 10.1002/cbin.11972

18512 Li J, Lv A, Chen M, Xu L, Huang H. Activating transcription factor 4 in erythroid development and [Formula: see text]-thalassemia: a powerful regulator with therapeutic potential Ann Hematol 2023 Oct 31 PubMed ID: 37906269 DOI: 10.1007/s00277-023-05508-8

18513 Shen F, Dassama LMK. Opportunities and challenges of protein-based targeted protein degradation Chem Sci 2023 14(32):8433-8447 PubMed ID: 37592990 DOI: 10.1039/d3sc02361c

18520 Ma L, Yang S, Peng Q, Zhang J, Zhang J. CRISPR/Cas9-based gene-editing technology for sickle cell disease Gene 2023 874:147480 PubMed ID: 37182559 DOI: 10.1016/j.gene.2023.147480

18522 Khiabani A, Kohansal MH, Keshavarzi A, Shahraki H, Kooshesh M, Karimzade M, Gholizadeh Navashenaq J. CRISPR/Cas9, a promising approach for the treatment of β-thalassemia: a systematic review Mol Genet Genomics 2023 298(1):1-11 PubMed ID: 36403178 DOI: 10.1007/s00438-022-01978-z

18527 Kargutkar N, Hariharan P, Nadkarni A. Dynamic interplay of microRNA in diseases and therapeutic Clin Genet 2023 103(3):268-276 PubMed ID: 36310341 DOI: 10.1111/cge.14256

18528 Paschoudi K, Yannaki E, Psatha N. Precision Editing as a Therapeutic Approach for β-Hemoglobinopathies Int J Mol Sci 2023 24(11):9527 PubMed ID: 37298481 DOI: 10.3390/ijms24119527

18540 Kraus C, Sontheimer EJ. Applications of Anti-CRISPR Proteins in Genome Editing and Biotechnology J Mol Biol 2023 435(13):168120 PubMed ID: 37100169 DOI: 10.1016/j.jmb.2023.168120

18544 Sahu S, Poplawska M, Lim SH, Dutta D. CRISPR-based precision medicine for hematologic disorders: Advancements, challenges, and prospects Life Sci 2023 333:122165 PubMed ID: 37832631 DOI: 10.1016/j.lfs.2023.122165

18545 Voloshin N, Tyurin-Kuzmin P, Karagyaur M, Akopyan Z, Kulebyakin K. Practical Use of Immortalized Cells in Medicine: Current Advances and Future Perspectives Int J Mol Sci 2023 24(16):12716 PubMed ID: 37628897 DOI: 10.3390/ijms241612716

18546 Finotti A, Gambari R. Combined approaches for increasing fetal hemoglobin (HbF) and de novo production of adult hemoglobin (HbA) in erythroid cells from β-thalassemia patients: treatment with HbF inducers and CRISPR-Cas9 based genome editing Front Genome Ed 2023 5:1204536 PubMed ID: 37529398 DOI: 10.3389/fgeed.2023.1204536

18547 Lu X, Zhang M, Li G, Zhang S, Zhang J, Fu X, Sun F. Applications and Research Advances in the Delivery of CRISPR/Cas9 Systems for the Treatment of Inherited Diseases Int J Mol Sci 2023 24(17):13202 PubMed ID: 37686009 DOI: 10.3390/ijms241713202

18548 Weiner L, Brissette JL. Finding meaning in chaos: a selection signature for functional interactions and its use in molecular biology FEBS J 2023 290(16):3914-3927. PubMed ID: 35653424 DOI: 10.1111/febs.16542

17392 Takase S, Hiroyama T, Shirai F, Maemoto Y, Nakata A, Arata M, Matsuoka S, Sonoda T, Niwa H, Sato S, Umehara T, Shirouzu M, Nishigaya Y, Sumiya T, Hashimoto N, Namie R, Usui M, Ohishi T, Ohba SI, Kawada M, Hayashi Y, Harada H, Yamaguchi T, Shinkai Y, Nakamura Y, Yoshida M, Ito A. A specific G9a inhibitor unveils BGLT3 lncRNA as a universal mediator of chemically induced fetal globin gene expression Nat Commun 2023 14(1):23. PubMed ID: 36635268 DOI: 10.1038/s41467-022-35404-0

17721 Soboleva S, Kurita R, Kajitani N, Åkerstrand H, Miharada K. Establishment of an immortalized human erythroid cell line sustaining differentiation potential without inducible gene expression system Hum Cell 2022 35(1):408-417 PubMed ID: 34817797 DOI: 10.1007/s13577-021-00652-7

18352 Dulmovits BM, Tang Y, Papoin J, He M, Li J, Yang H, Addorisio ME, Kennedy L, Khan M, Brindley E, Ashley RJ, Ackert-Bicknell C, Hale J, Kurita R, Nakamura Y, Diamond B, Barnes BJ, Hermine O, Gallagher PG, Steiner LA, Lipton JM, Taylor N, Mohandas N, Andersson U, Al-Abed Y, Tracey KJ, Blanc L. HMGB1-mediated restriction of EPO signaling contributes to anemia of inflammation Blood 2022 139(21):3181-3193. PubMed ID: 35040907 DOI: 10.1182/blood.2021012048

18353 Ravi NS, Wienert B, Wyman SK, Bell HW, George A, Mahalingam G, Vu JT, Prasad K, Bandlamudi BP, Devaraju N, Rajendiran V, Syedbasha N, Pai AA, Nakamura Y, Kurita R, Narayanasamy M, Balasubramanian P, Thangavel S, Marepally S, Velayudhan SR, Srivastava A, DeWitt MA, Crossley M, Corn JE, Mohankumar KM. Identification of novel HPFH-like mutations by CRISPR base editing that elevate the expression of fetal hemoglobin Elife 2022 11:e65421 PubMed ID: 35147495 DOI: 10.7554/eLife.65421

18354 Wang B, Wang C, Wan Y, Gao J, Ma Y, Zhang Y, Tong J, Zhang Y, Liu J, Chang L, Xu C, Shen B, Chen Y, Jiang E, Kurita R, Nakamura Y, Lim KC, Engel JD, Zhou J, Cheng T, Zhu X, Zhu P, Shi L. Decoding the pathogenesis of Diamond-Blackfan anemia using single-cell RNA-seq Cell Discov 2022 8(1):41 PubMed ID: 35534476 DOI: 10.1038/s41421-022-00389-z

18355 Lu D, Xu Z, Peng Z, Yang Y, Song B, Xiong Z, Ma Z, Guan H, Chen B, Nakamura Y, Zeng J, Liu N, Sun X, Chen D. Induction of Fetal Hemoglobin by Introducing Natural Hereditary Persistence of Fetal Hemoglobin Mutations in the γ-Globin Gene Promoters for Genome Editing Therapies for β-Thalassemia Front Genet 2022 13:881937. PubMed ID: 35656314 DOI: 10.3389/fgene.2022.881937

18356 Bagchi A, Devaraju N, Chambayil K, Rajendiran V, Venkatesan V, Sayed N, Pai AA, Nath A, David E, Nakamura Y, Balasubramanian P, Srivastava A, Thangavel S, Mohankumar KM, Velayudhan SR. Erythroid lineage-specific lentiviral RNAi vectors suitable for molecular functional studies and therapeutic applications Sci Rep 2022 12(1):14033 PubMed ID: 35982069 DOI: 10.1038/s41598-022-13783-0

18357 Ochi T, Fujiwara T, Ono K, Suzuki C, Nikaido M, Inoue D, Kato H, Onodera K, Ichikawa S, Fukuhara N, Onishi Y, Yokoyama H, Nakamura Y, Harigae H. Exploring the mechanistic link between SF3B1 mutation and ring sideroblast formation in myelodysplastic syndrome Sci Rep 2022 12(1):14562 PubMed ID: 36028755 DOI: 10.1038/s41598-022-18921-2

18366 Koya Ono, Tohru Fujiwara, Hiroki Shima, Hironari Nishizawa, Chie Suzuki, Noriyuki Takahashi, Hiroki Kato, Koichi Onodera, Satoshi Ichikawa, Noriko Fukuhara, Yasushi Onishi, Hisayuki Yokoyama, Yukio Nakamura, Kazuhiko Igarashi, Hideo Harigae Altered Transcription By GATA1 Impairs Autophagy and Prevents Ferroptosis in X-Linked Sideroblastic Anemia Blood 2022 140:2467-2468 DOI: 10.1182/blood-2022-163073

18372 King R, Gallagher PJ, Khoriaty R. The congenital dyserythropoieitic anemias: genetics and pathophysiology Curr Opin Hematol 2022 29(3):126-136 PubMed ID: 35441598 DOI: 10.1097/MOH.0000000000000697

18385 Han Y, Tan X, Jin T, Zhao S, Hu L, Zhang W, Kurita R, Nakamura Y, Liu J, Li D, Zhang Z, Fang X, Huang S. CRISPR/Cas9-based multiplex genome editing of BCL11A and HBG efficiently induces fetal hemoglobin expression Eur J Pharmacol 2022 918:174788 PubMed ID: 35093321 DOI: 10.1016/j.ejphar.2022.174788

18401 Eccleston A. HIF1α reboots fetal haemoglobin production Nat Rev Drug Discov 2022 21(12):878 PubMed ID: 36307651 DOI: 10.1038/d41573-022-00179-y

18494 Feldman TP, Egan ES. Uncovering a Cryptic Site of Malaria Pathogenesis: Models to Study Interactions Between Plasmodium and the Bone Marrow Front Cell Infect Microbiol 2022 12:917267 PubMed ID: 35719356 DOI: 10.3389/fcimb.2022.917267

18517 Mukherjee M, Rahaman M, Ray SK, Shukla PC, Dolai TK, Chakravorty N. Revisiting fetal hemoglobin inducers in beta-hemoglobinopathies: a review of natural products, conventional and combinatorial therapies Mol Biol Rep 2022 49(3):2359-2373 PubMed ID: 34822068 DOI: 10.1007/s11033-021-06977-8

18523 Tetard M, Peterson NA, Egan ES. Erythrocyte-Plasmodium interactions: genetic manipulation of the erythroid lineage Curr Opin Microbiol 2022 70:102221 PubMed ID: 36242898 DOI: 10.1016/j.mib.2022.102221

18525 Boutin J, Cappellen D, Rosier J, Amintas S, Dabernat S, Bedel A, Moreau-Gaudry F. ON-Target Adverse Events of CRISPR-Cas9 Nuclease: More Chaotic than Expected CRISPR J 2022 5(1):19-30 PubMed ID: 35099280 DOI: 10.1089/crispr.2021.0120

18529 Chauhan W, Shoaib S, Fatma R, Zaka-Ur-Rab Z, Afzal M. Beta-thalassemia and the advent of new interventions beyond transfusion and iron chelation Br J Clin Pharmacol 2022 88(8):3610-3626 PubMed ID: 35373382 DOI: 10.1111/bcp.15343

18531 Reshetnikov VV, Chirinskaite AV, Sopova JV, Ivanov RA, Leonova EI. Translational potential of base-editing tools for gene therapy of monogenic diseases Front Bioeng Biotechnol 2022 10:942440 PubMed ID: 36032737 DOI: 10.3389/fbioe.2022.942440

18532 Ding J, Frantzeskos A, Orozco G. Functional interrogation of autoimmune disease genetics using CRISPR/Cas9 technologies and massively parallel reporter assays Semin Immunopathol 2022 44(1):137-147 PubMed ID: 34508276 DOI: 10.1007/s00281-021-00887-4

18535 Germino-Watnick P, Hinds M, Le A, Chu R, Liu X, Uchida N. Hematopoietic Stem Cell Gene-Addition/Editing Therapy in Sickle Cell Disease Cells 2022 11(11):1843 PubMed ID: 35681538 DOI: 10.3390/cells11111843

18538 Taha EA, Lee J, Hotta A. Delivery of CRISPR-Cas tools for in vivo genome editing therapy: Trends and challenges J Control Release 2022 342:345-361 PubMed ID: 35026352 DOI: 10.1016/j.jconrel.2022.01.013

18541 Rahimmanesh I, Boshtam M, Kouhpayeh S, Khanahmad H, Dabiri A, Ahangarzadeh S, Esmaeili Y, Bidram E, Vaseghi G, Haghjooy Javanmard S, Shariati L, Zarrabi A, Varma RS. Gene Editing-Based Technologies for Beta-hemoglobinopathies Treatment Biology (Basel) 2022 11(6):862 PubMed ID: 35741383 DOI: 10.3390/biology11060862

18543 Li Z, Zhou P, Kwon E, Fitzgerald KA, Weng Z, Zhou C. Flnc: Machine Learning Improves the Identification of Novel Long Noncoding RNAs from Stand-Alone RNA-Seq Data Noncoding RNA 2022 8(5):70 PubMed ID: 36287122 DOI: 10.3390/ncrna8050070

13945 Papasavva PL, Patsali P, Loucari CC, Kurita R, Nakamura Y, Kleanthous M, Lederer CW. CRISPR Editing Enables Consequential Tag-Activated MicroRNA-Mediated Endogene Deactivation Int J Mol Sci 2022 23(3):1082 PubMed ID: 35163006 DOI: 10.3390/ijms23031082

20678 Ono K, Fujiwara T, Saito K, Nishizawa H, Takahashi N, Suzuki C, Ochi T, Kato H, Ishii Y, Onodera K, Ichikawa S, Fukuhara N, Onishi Y, Yokoyama H, Yamada R, Nakamura Y, Igarashi K, Harigae H. Congenital sideroblastic anemia model due to ALAS2 mutation is susceptible to ferroptosis. Sci Rep 2022 12(1):9024 PubMed ID: 35637209 DOI: 10.1038/s41598-022-12940-9

20811 Tingting Jin, Zhaojun Zhang, Yuanyuan Han, Di Li, Juan Liu, Minmin Jiang, Junwei Zhu, Ryo Kurita, Yukio Nakamura, Fangfang Hu, Yongjie Xu, Xiangdong Fang, Shengwen Huang , and Zhaolin Sun Transmembrane Protein ANTXR1 Regulates γ-Globin Expression by Targeting the Wnt/ β-Catenin Signaling Pathway. Journal of Immunology Research 2022 PubMed ID: 35942209 DOI: 10.1155/2022/8440422

20830 Tingting Jin, Zhaojun Zhang, Yuanyuan Han, Di Li, Juan Liu, Minmin Jiang, Ryo Kurita, Yukio Nakamura, Fangfang Hu, Xiangdong Fang, Shengwen Huang, and Zhaolin Sun ANTXR1 Regulates Erythroid Cell Proliferation and Differentiation through wnt/β-Catenin Signaling Pathway In Vitro and in Hematopoietic Stem Cell Disease Markers 2022 15 PubMed ID: 36065334 DOI: 10.1155/2022/1226697

17722 Soboleva S, Kurita R, Ek F, Åkerstrand H, Silvério-Alves R, Olsson R, Nakamura Y, Miharada K. Identification of potential chemical compounds enhancing generation of enucleated cells from immortalized human erythroid cell lines Commun Biol 2021 4(1):677 PubMed ID: 34083702 DOI: 10.1038/s42003-021-02202-1

18339 Mingoia M, Caria CA, Ye L, Asunis I, Marongiu MF, Manunza L, Sollaino MC, Wang J, Cabriolu A, Kurita R, Nakamura Y, Cucca F, Kan YW, Marini MG, Moi P. Induction of therapeutic levels of HbF in genome-edited primary β0 39-thalassaemia haematopoietic stem and progenitor cells Br J Haematol 2021 192(2):395-404 PubMed ID: 33216968 DOI: 10.1111/bjh.17167

18340 Ishii S, Suzuki T, Wakahashi K, Asada N, Kawano Y, Kawano H, Sada A, Minagawa K, Nakamura Y, Mizuno S, Takahashi S, Matsui T, Katayama Y. タイトル FGF-23 from erythroblasts promotes hematopoietic progenitor mobilization Blood 2021 137(11):1457-1467 PubMed ID: 33512467 DOI: 10.1182/blood.2020007172

18341 Breda L, Ghiaccio V, Tanaka N, Jarocha D, Ikawa Y, Abdulmalik O, Dong A, Casu C, Raabe TD, Shan X, Danet-Desnoyers GA, Doto AM, Everett J, Bushman FD, Radaelli E, Assenmacher CA, Tarrant JC, Hoepp N, Kurita R, Nakamura Y, Guzikowski V, Smith-Whitley K, Kwiatkowski JL, Rivella S. Lentiviral vector ALS20 yields high hemoglobin levels with low genomic integrations for treatment of beta-globinopathies Mol Ther . 2021 Apr 7;29(4):1625-1638 2021 Mol Ther . 2021 Apr 7;29(4):1625-1638 PubMed ID: 33515514 DOI: 10.1016/j.ymthe.2020.12.036

18342 Bao X, Zhang X, Wang L, Wang Z, Huang J, Zhang Q, Ye Y, Liu Y, Chen D, Zuo Y, Liu Q, Xu P, Huang B, Fang J, Lao J, Feng X, Li Y, Kurita R, Nakamura Y, Yu W, Ju C, Huang C, Mohandas N, Li D, Zhao C, Xu X. Epigenetic inactivation of ERF reactivates γ-globin expression in β-thalassemia Am J Hum Genet 2021 108(4):709-721 PubMed ID: 33735615 DOI: 10.1016/j.ajhg.2021.03.005

18343 Bagchi A, Nath A, Thamodaran V, Ijee S, Palani D, Rajendiran V, Venkatesan V, Datari P, Pai AA, Janet NB, Balasubramanian P, Nakamura Y, Srivastava A, Mohankumar KM, Thangavel S, Velayudhan SR. Direct Generation of Immortalized Erythroid Progenitor Cell Lines from Peripheral Blood Mononuclear Cells Cells 2021 10(3):523. PubMed ID: 33804564 DOI: 10.3390/cells10030523

18344 Liang Y, Zhang X, Liu Y, Wang L, Ye Y, Tan X, Pu J, Zhang Q, Bao X, Wei X, Li D, Kurita R, Nakamura Y, Li D, Xu X. GATA zinc finger domain-containing protein 2A (GATAD2A) deficiency reactivates fetal haemoglobin in patients with β-thalassaemia through impaired formation of methyl-binding domain protein 2 (MBD2)-containing nucleosome remodelling and deacetylation (NuRD) complex Br J Haematol 2021 193(6):1220-1227 PubMed ID: 33997955 DOI: 10.1111/bjh.17511

18345 Pires Lourenco S, Jarocha D, Ghiaccio V, Guerra A, Abdulmalik O, La P, Zezulin A, Smith-Whitley K, Kwiatkowski JL, Guzikowski V, Nakamura Y, Raabe T, Breda L, Rivella S. Inclusion of a short hairpin RNA targeting BCL11A into a β-globin expressing vector allows concurrent synthesis of curative adult and fetal hemoglobin Haematologica 2021 106(10):2740-2745 PubMed ID: 34047176 DOI: 10.3324/haematol.2020.276634

18346 Jeffery NN, Davidson C, Peslak SA, Kingsley PD, Nakamura Y, Palis J, Bulger M. Histone H2A.X phosphorylation and Caspase-Initiated Chromatin Condensation in late-stage erythropoiesis Epigenetics Chromatin 2021 14(1):37. PubMed ID: 34330317 DOI: 10.1186/s13072-021-00408-5

18347 Li X, Chen M, Liu B, Lu P, Lv X, Zhao X, Cui S, Xu P, Nakamura Y, Kurita R, Chen B, Huang DCS, Liu DP, Liu M, Zhao Q. Transcriptional silencing of fetal hemoglobin expression by NonO Nucleic Acids Res 2021 49(17):9711-9723. PubMed ID: 34379783 DOI: 10.1093/nar/gkab671

18348 Daniels DE, Ferguson DCJ, Griffiths RE, Trakarnsanga K, Cogan N, MacInnes KA, Mordue KE, Andrienko T, Ferrer-Vicens I, Ramos Jiménez D, Lewis PA, Wilson MC, Canham MA, Kurita R, Nakamura Y, Anstee DJ, Frayne J. Reproducible immortalization of erythroblasts from multiple stem cell sources provides approach for sustainable RBC therapeutics Mol Ther Methods Clin Dev 2021 22:26-39 PubMed ID: 34485592 DOI: 10.1016/j.omtm.2021.06.002

18349 Himadewi P, Wang XQD, Feng F, Gore H, Liu Y, Yu L, Kurita R, Nakamura Y, Pfeifer GP, Liu J, Zhang X. 3'HS1 CTCF binding site in human β-globin locus regulates fetal hemoglobin expression Elife 2021 10:e70557 PubMed ID: 34585664 DOI: 10.7554/eLife.70557

18351 Nath A, Rayabaram J, Ijee S, Bagchi A, Chaudhury AD, Roy D, Chambayil K, Singh J, Nakamura Y, Velayudhan SR. Comprehensive Analysis of microRNAs in Human Adult Erythropoiesis Cells 2021 10(11):3018 PubMed ID: 34831239 DOI: 10.3390/cells10113018

18398 Tumburu L, Thein SL. Targeting ZNF410 as a potential β-hemoglobinopathy therapy Nat Genet 2021 53(5):589-590 PubMed ID: 33859418 DOI: 10.1038/s41588-021-00817-y

18430 Cruz LJ, van Dijk T, Vepris O, Li TMWY, Schomann T, Baldazzi F, Kurita R, Nakamura Y, Grosveld F, Philipsen S, Eich C. PLGA-Nanoparticles for Intracellular Delivery of the CRISPR-Complex to Elevate Fetal Globin Expression in Erythroid Cells Biomaterials 2021 268:120580 PubMed ID: 33321292 DOI: 10.1016/j.biomaterials.2020.120580

18503 Demirci S, Leonard A, Essawi K, Tisdale JF. CRISPR-Cas9 to induce fetal hemoglobin for the treatment of sickle cell disease Mol Ther Methods Clin Dev 2021 23:276-285. PubMed ID: 34729375 DOI: 10.1016/j.omtm.2021.09.010

18516 Rao S, Yao Y, Bauer DE. Editing GWAS: experimental approaches to dissect and exploit disease-associated genetic variation Genome Med 2021 13(1):41 PubMed ID: 33691767 DOI: 10.1186/s13073-021-00857-3

18521 Frati G, Miccio A. Genome Editing for β-Hemoglobinopathies: Advances and Challenges J Clin Med 2021 10(3):482. PubMed ID: 33525591 DOI: 10.3390/jcm10030482

18524 Mettananda S. Genetic and Epigenetic Therapies for β-Thalassaemia by Altering the Expression of α-Globin Gene Front Genome Ed 2021 3:752278 PubMed ID: 34713267 DOI: 10.3389/fgeed.2021.752278

18526 Azhagiri MKK, Babu P, Venkatesan V, Thangavel S. Homology-directed gene-editing approaches for hematopoietic stem and progenitor cell gene therapy Stem Cell Res Ther 2021 12(1):500 PubMed ID: 34503562 DOI: 10.1186/s13287-021-02565-6

18533 Sharif SB, Zamani N, Chadwick BP. BAZ1B the Protean Protein Genes (Basel) 2021 ;12(10):1541 PubMed ID: 34680936 DOI: 10.3390/genes12101541

18539 Hariharan P, Nadkarni A. Insight of fetal to adult hemoglobin switch: Genetic modulators and therapeutic targets Blood Rev 2021 49:100823 PubMed ID: 33726930 DOI: 10.1016/j.blre.2021.100823

20087 Ma Y, Liu S, Gao J, Chen C, Zhang X, Yuan H, Chen Z, Yin X, Sun C, Mao Y, Zhou F, Shao Y, Liu Q, Xu J, Cheng L, Yu D, Li P, Yi P, He J, Geng G, Guo Q, Si Y, Zhao H, Li H, Banes GL, Liu H, Nakamura Y, Kurita R, Huang Y, Wang X, Wang F, Fang G, Engel JD, Shi L, Zhang YE, Yu J. Genome-wide analysis of pseudogenes reveals HBBP1's human-specific essentiality in erythropoiesis and implication in β-thalassemia. Dev Cell 2021 56(4):478-493.e11 PubMed ID: 33476555 DOI: 10.1016/j.devcel.2020.12.019

20294 Ma SP, Xi HR, Gao XX, Yang JM, Kurita R, Nakamura Y, Song XM, Chen HY, Lu DR. Long noncoding RNA HBBP1 enhances γ-globin expression through the ETS transcription factor ELK1. Biochem Biophys Res Commun 2021 552:157-163 PubMed ID: 33744764 DOI: 10.1016/j.bbrc.2021.03.051

15517 Papasavva PL, Papaioannou NY, Patsali P, Kurita R, Nakamura Y, Sitarou M, Christou S, Kleanthous M, Lederer CW. Distinct miRNA Signatures and Networks Discern Fetal from Adult Erythroid Differentiation and Primary from Immortalized Erythroid Cells Int J Mol Sci 2021 22(7):3626. PubMed ID: 33807258 DOI: 10.3390/ijms22073626

15638 King R, Lin Z, Balbin-Cuesta G, Myers G, Friedman A, Zhu G, McGee B, Saunders TL, Kurita R, Nakamura Y, Engel JD, Reddy P, Khoriaty R. SEC23A rescues SEC23B-deficient congenital dyserythropoietic anemia type II Sci Adv 2021 7(48):eabj5293 PubMed ID: 34818036 DOI: 10.1126/sciadv.abj5293

16042 Yannis Hara, Dipti Gupta, Courtney Mercadante, Kim Alving, Yukio Nakamura, Sriram Krishnamoorthy, Alexandra Hicks, Melanie Demers Heme-Regulated eIF2α Kinase (HRI) Inhibition Decreases PKR Activation in HUDEP2 Cells Blood 2021 138:2035-2035 DOI: 10.1182/blood-2021-144679

16066 Wessels MW, Cnossen MH, van Dijk TB, Gillemans N, Schmidt KLJ, van Lom K, Vinjamur DS, Coyne S, Kurita R, Nakamura Y, de Man SA, Pfundt R, Azmani Z, Brouwer RWW, Bauer DE, van den Hout MCGN, van IJcken WFJ, Philipsen S. Molecular analysis of the erythroid phenotype of a patient with BCL11A haploinsufficiency Blood Adv 2021 5(9):2339-2349 PubMed ID: 33938942 DOI: 10.1182/bloodadvances.2020003753

18338 Zhang X, Zhu B, Chen L, Xie L, Yu W, Wang Y, Li L, Yin S, Yang L, Hu H, Han H, Li Y, Wang L, Chen G, Ma X, Geng H, Huang W, Pang X, Yang Z, Wu Y, Siwko S, Kurita R, Nakamura Y, Yang L, Liu M, Li D. Dual base editor catalyzes both cytosine and adenine base conversions in human cells Nat Biotechnol 2020 38(7):856-860 PubMed ID: 32483363 DOI: 10.1038/s41587-020-0527-y

18364 Dipti Gupta, Samuel Lessard, Shujia Dai, Yukio Nakamura, Alexandra Hicks, Thomas K Harris, Sriram Krishnamoorthy Genetic Silencing of KEAP1 Induces NRF2 Mediated Oxidative Stress Pathway in Human Erythroid Cells Blood 2020 136:8-9 DOI: 10.1182/blood-2020-139730

18365 Dipti Gupta, Yannis Hara, Samuel Lessard, Sarah Sturtevant, Yukio Nakamura, Sriram Krishnamoorthy, Melanie Demers, Alexandra Hicks Catalytic Activity of Heme-Regulated eIF2 Alpha Kinase (HRI) Regulates Fetal Hemoglobin Blood 2020 136:7-7 DOI: 10.1182/blood-2020-139991

18445 Trakarnsanga K, Tipgomut C, Metheetrairut C, Wattanapanitch M, Khuhapinant A, Poldee S, Kurita R, Nakamura Y, Srisawat C, Frayne J. Generation of an immortalised erythroid cell line from haematopoietic stem cells of a haemoglobin E/β-thalassemia patient Sci Rep 2020 10(1):16798 PubMed ID: 33033327 DOI: 10.1038/s41598-020-73991-4

18498 Demirci S, Leonard A, Tisdale JF. Genome editing strategies for fetal hemoglobin induction in beta-hemoglobinopathies Hum Mol Genet 2020 29(R1):R100-R106 PubMed ID: 32406490 DOI: 10.1093/hmg/ddaa088

18501 Steinberg MH. Fetal Hemoglobin in Sickle Hemoglobinopathies: High HbF Genotypes and Phenotypes J Clin Med 2020 9(11):3782 PubMed ID: 33238542 DOI: 10.3390/jcm9113782

18507 Venkatesan V, Srinivasan S, Babu P, Thangavel S. Manipulation of Developmental Gamma-Globin Gene Expression: an Approach for Healing Hemoglobinopathies Mol Cell Biol 2020 41(1):e00253-20 PubMed ID: 33077498 DOI: 10.1128/MCB.00253-20

18536 Tasca F, Wang Q, Gonçalves MAFV. Adenoviral Vectors Meet Gene Editing: A Rising Partnership for the Genomic Engineering of Human Stem Cells and Their Progeny Cells 2020 9(4):953 PubMed ID: 32295080 DOI: 10.3390/cells9040953

6747 Daniels DE, Downes DJ, Ferrer-Vicens I, Ferguson DCJ, Singleton BK, Wilson MC, Trakarnsanga K, Kurita R, Nakamura Y, Anstee DJ, Frayne J Comparing the two leading erythroid lines BEL-A and HUDEP-2. Haematologica 2020 105(8):e389-e394 PubMed ID: 31753923 DOI: 10.3324/haematol.2019.229211

6796 Balogh P, Adelman ER, Pluvinage JV, Capaldo BJ, Freeman KC, Singh S, Elagib KE, Nakamura Y, Kurita R, Sashida G, Zunder ER, Li H, Gru AA, Price EA, Schrier SL, Weissman IL, Figueroa ME, Pang WW, Goldfarb AN. RUNX3 levels in human hematopoietic progenitors are regulated by aging and dictate erythroid-myeloid balance. Haematologica 2020 105(4):905-913 PubMed ID: 31171641 DOI: 10.3324/haematol.2018.208918

8959 Liren Wang, Linxi Li, Yanlin Ma, Handan Hu 1, Qi Li, Yang Yang, Wenbang Liu, Shuming Yin, Wei Li, Bin Fu, Ryo Kurita, Yukio Nakamura, Mingyao Liu, Yongrong Lai, Dali Li Reactivation of γ-Globin Expression Through Cas9 or Base Editor to Treat β-Hemoglobinopathies Cell Res 2020 30 (3), 276-278 PubMed ID: 31911671 DOI: 10.1038/s41422-019-0267-z

8964 Jun Zhan, Maria Johnson Irudayam, Yukio Nakamura, Ryo Kurita, Arthur W Nienhuis High Level of Fetal-Globin Reactivation by Designed Transcriptional Activator-Like Effector Blood Adv 2020 4 (4), 687-695 PubMed ID: 32084259 DOI: 10.1182/bloodadvances.2019000482

9631 Morgan RA, Ma F, Unti MJ, Brown D, Ayoub PG, Tam C, Lathrop L, Aleshe B, Kurita R, Nakamura Y, Senadheera S, Wong RL, Hollis RP, Pellegrini M, Kohn DB. Creating New β-globin-Expressing Lentiviral Vectors by High-Resolution Mapping of Locus Control Region Enhancer Sequences Mol Ther Methods Clin Dev 2020 17:999-1013 PubMed ID: 32426415 DOI: 10.1016/j.omtm.2020.04.006

18519 Demirci S, Leonard A, Haro-Mora JJ, Uchida N, Tisdale JF. CRISPR/Cas9 for Sickle Cell Disease: Applications, Future Possibilities, and Challenges Adv Exp Med Biol 2019 1144:37-52 PubMed ID: 30715679 DOI: 10.1007/5584_2018_331

18542 Katsantoni E. Omics Studies in Hemoglobinopathies Mol Diagn Ther 2019 23(2):223-234 PubMed ID: 30712217 DOI: 10.1007/s40291-019-00386-1

6743 Chung JE, Magis W, Vu J, Heo SJ, Wartiovaara K, Walters MC, Kurita R, Nakamura Y, Boffelli D, Martin DIK, Corn JE, DeWitt MA CRISPR-Cas9 interrogation of a putative fetal globin repressor in human erythroid cells. PLoS One 2019 14(1):e0208237 PubMed ID: 30645582 DOI: 10.1371/journal.pone.0208237

6744 Couch T, Murphy Z, Getman M, Kurita R, Nakamura Y, Steiner LA. Human erythroblasts with c-Kit activating mutations have reduced cell culture costs and remain capable of terminal maturation. Exp Hematol 2019 19-24.e4 PubMed ID: 31004744 DOI: 10.1016/j.exphem.2019.04.001

6752 Svetlana Soboleva , Ryo Kurita, PhD , Fredrik Ek, PhD , Rita Alves , Roger Olsson, PhD , Yukio Nakamura, MD PhD , Kenichi Miharada, PhD Identification of Potential Chemical Compounds Able to Trigger Enucleation of Immortalized Human Erythroid Cell Lines Blood 2019 134 (Supplement_1): 951 DOI: 10.1182/blood-2019-128427

6759 Koya Ono, MD , Tohru Fujiwara, MD PhD , Kei Saito, MD PhD , Chie Suzuki , Noriyuki Takahashi , Koichi Onodera, MD PhD , Satoshi Ichikawa, MD PhD , Noriko Fukuhara, MD PhD , Yasushi Onishi, MD PhD , Hisayuki Yokoyama, MD PhD , Yukio Nakamura, MD PhD , Hideo Harigae, MD PhD Molecular Characterization and Novel Therapeutic Strategy for X-Linked Sideroblastic Anemia Associated with ALAS2 Missense Variants Blood 2019 134 (Supplement_1): 3530 DOI: 10.1182/blood-2019-124810

6763 Ingrid Grazielle Sousa, BSc, MSc , Priscila Keiko Matsumoto Martin, PhD , Dulcinéia Martins de Albuquerque, BSc, PhD , Carolina Lanaro, BSc, PhD , Ryo Kurita, PhD , Yukio Nakamura, MD PhD , Fernando Ferreira Costa, MD PhD Knockdown of HNF4A Gene Increases Fetal Hemoglobin Synthesis in Hudep-2 Blood 2019 134 (Supplement_1): 968 DOI: 10.1182/blood-2019-130920

6768 Tohru Fujiwara, MD PhD , Chie Suzuki , Tetsuro Ochi , Koya Ono, MD , Kei Saito , Noriko Fukuhara, MD PhD , Yasushi Onishi, MD PhD , Hisayuki Yokoyama, MD PhD , Shinichi Fujimaki , Yukio Nakamura, MD PhD , Hideo Harigae, MD PhD Characterization of Congenital Sideroblastic Anemia Model Due to ABCB7 Defects: How Do Defects in Iron-Sulfur Cluster Metabolism Lead to Ring Sideroblast Formation? Blood 2019 134 (Supplement_1): 2232 DOI: 10.1182/blood-2019-123918

6770 Melanie Demers, PhD , Sarah Sturtevant, BSc , Kevin Guertin, PhD , Dipti Gupta , Kunal Desai, MS , Benjamin Vieira, BS , Alexandra Hicks, PhD , Ayman Ismail, PhD , Yukio Nakamura, MD PhD , Bronner Goncalves, MD PhD , Giuseppe Di Caprio, PhD , Ethan Schonbrun, PhD , Scott Hansen, BS , Martin K. Safo, PhD , David K. Wood, PhD , John M. Higgins, MD PhD , David R. Light, PhD MetAP2 Inhibition Modifies Hemoglobin S (HbS) to Delay Polymerization and Improve Blood Flow in Sickle Cell Disease Blood 2019 134 (Supplement_1): 2260 DOI: 10.1182/blood-2019-128727

6771 Dipti Gupta , Sarah Sturtevant, BSc , Benjamin Vieira, BS , Yukio Nakamura, MD PhD , Sriram Krishnamoorthy, PhD , Melanie Demers, PhD Characterization of a Genetically Engineered HUDEP2 Cell Line Harboring a Sickle Cell Disease Mutation As a Potential Research Tool for Preclinical Sickle Cell Disease Drug Discovery Blood 2019 134 (Supplement_1): 3559 DOI: 10.1182/blood-2019-127330

6776 Orkin SH, Bauer DE. Emerging Genetic Therapy for Sickle Cell Disease Annu Rev Med 2019 70:257-271 PubMed ID: 30355263 DOI: 10.1146/annurev-med-041817-125507

6789 Yu X, Azzo A, Bilinovich SM, Li X, Dozmorov M, Kurita R, Nakamura Y, Williams DC Jr, Ginder GD. Disruption of the MBD2-NuRD complex but not MBD3-NuRD induces high level HbF expression in human adult erythroid cells. Haematologica 2019 104(12):2361-2371 PubMed ID: 31004025 DOI: 10.3324/haematol.2018.210963

6793 Kurita R, Funato K, Abe T, Watanabe Y, Shiba M, Tadokoro K, Nakamura Y, Nagai T, Satake M Establishment and characterization of immortalized erythroid progenitor cell lines derived from a common cell source. Exp Hematol 2019 69:11-16 PubMed ID: 30326248 DOI: 10.1016/j.exphem.2018.10.005

6799 Sher F, Hossain M, Seruggia D, Schoonenberg VAC, Yao Q, Cifani P, Dassama LMK, Cole MA, Ren C, Vinjamur DS, Macias-Trevino C, Luk K, McGuckin C, Schupp PG, Canver MC, Kurita R, Nakamura Y, Fujiwara Y, Wolfe SA, Pinello L, Maeda T, Kentsis A, Orkin SH, Bauer DE Rational targeting of a NuRD subcomplex guided by comprehensive in situ mutagenesis. Nat Genet 2019 51(7):1149-1159 PubMed ID: 31253978 DOI: 10.1038/s41588-019-0453-4

6811 Damien J. Downes, Ron Schwessinger, Stephanie J. Hill, Lea Nussbaum, Caroline Scott, Matthew E. Gosden, Priscila P. Hirschfeld, Jelena M. Telenius, Chris Q. Eijsbouts, Simon J. McGowan, Antony J. Cutler, Jon Kerry, Jessica L. Davies, Calliope A. Dendrou, Jamie R.J. Inshaw, Martin S.C. Larke, A. Marieke Oudelaar, Yavor Bozhilov, Andrew J. King, Richard C. Brown, Maria C. Suciu, James O.J. Davies, Philip Hublitz, Chris Fisher, Ryo Kurita, Yukio Nakamura, Gerton Lunter, Stephen Taylor, Veronica J. Buckle, John A. Todd, Douglas R. Higgs, & Jim R. Hughes. An integrated platform to systematically identify causal variants and genes for polygenic human traits bioRxiv 2019 DOI: 10.1101/813618

6815 Damena D, Denis A, Golassa L, Chimusa ER. Genome-wide association studies of severe P. falciparum malaria susceptibility: progress, pitfalls and prospects. BMC Med Genomics 2019 12(1):120 PubMed ID: 31409341 DOI: 10.1186/s12920-019-0564-x

6821 Finotti A, Fabbri E, Lampronti I, Gasparello J, Borgatti M, Gambari R. MicroRNAs and Long Non-coding RNAs in Genetic Diseases. Mol Diagn Ther 2019 23(2):155-171 PubMed ID: 30610665 DOI: 10.1007/s40291-018-0380-6

8953 Gabriella E Martyn, Beeke Wienert, Ryo Kurita, Yukio Nakamura, Kate G R Quinlan, Merlin Crossley A Natural Regulatory Mutation in the Proximal Promoter Elevates Fetal globin Expression by Creating a De Novo GATA1 Site Blood 2019 133 (8), 852-856 PubMed ID: 30617196 DOI: 10.1182/blood-2018-07-863951

9888 Dipti Gupta, Samuel Lessard, PhD, Nancy Moore, Jicheng Duan, PhD, Yukio Nakamura, MD PhD, Feng-Chun Yang, MD PhD, Alexandra Hicks, PhD, David R. Light, PhD, Sriram Krishnamoorthy, PhD Genetic Activation of NRF2 By KEAP1 Inhibition Induces Fetal Hemoglobin Expression and Triggers Anti-Oxidant Stress Response in Erythroid Cells Blood 2019 134 (Supplement_1): 210 DOI: 10.1182/blood-2019-129479

18361 Mir Hossain, Falak Sher, Davide Seruggia, Vivien Schoonenberg, Mitchel A. Cole, Paolo Cifani, Laura Dassama, Claudio Macias-Trevino, Matthew C. Canver, Ryo Kurita, Yukio Nakamura, Luca Pinello, Takahiro Maeda, Alex Kentsis, Stuart H. Orkin, Daniel E. Bauer Rational Targeting of a NuRD Sub-Complex for Fetal Hemoglobin Induction Following Comprehensive in Situ Mutagenesis Blood 2018 132:2342-2342 DOI: 10.1182/blood-2018-99-117043

18362 Priscila Keiko Matsumoto Martin, Dulcinéia Martins de Albuquerque, Carolina Lanaro, Ryo Kurita, Yukio Nakamura, Fernando F. Costa A Single -195 C < G HBG1 Promoter Mediated By CRISPR/Cas9 Genome Editing Induces Fetal Hemoglobin Synthesis in Hudep-2 Blood 2018 132:3481-3481 DOI: 10.1182/blood-2018-99-118534

18363 Bin Sun, Valerie Miller, Karin P Potoka, Yukio Nakamura, Mark T Gladwin, Grant C. Bullock Soluble Guanylate Cyclase Modulators Effect Fetal Hemoglobin Levels through a Non-cGMP-Protein Kinase G Signaling Pathway Blood 2018 132:1060-1060 DOI: 10.1182/blood-2018-99-120306

18515 Philipsen S, Hardison RC. Evolution of hemoglobin loci and their regulatory elements Blood Cells Mol Dis 2018 70:2-12 PubMed ID: 28811072 DOI: 10.1016/j.bcmd.2017.08.001

18537 Thein SL. Molecular basis of β thalassemia and potential therapeutic targets Blood Cells Mol Dis 2018 70:54-65 PubMed ID: 28651846 DOI: 10.1016/j.bcmd.2017.06.001

18549 Zhang J, Yan J, Zeng F. Recent Progress on Genetic Diagnosis and Therapy for β-Thalassemia in China and Around the World Hum Gene Ther 2018 29(2):197-203 PubMed ID: 29357712 DOI: 10.1089/hum.2017.228

4404 Moir-Meyer G, Cheong PL, Olijnik AA, Brown J, Knight S, King A, Kurita R, Nakamura Y, Gibbons RJ, Higgs DR, Buckle VJ, Babbs C. Robust CRISPR/Cas9 Genome Editing of the HUDEP-2 Erythroid Precursor Line Using Plasmids and Single-Stranded Oligonucleotide Donors Methods Protoc 2018 1(3) PubMed ID: 31164570 DOI: 10.3390/mps1030028

4247 Liu J, Li Y, Tong J, Gao J, Guo Q, Zhang L, Wang B, Zhao H, Wang H, Jiang E, Kurita R, Nakamura Y, Tanabe O, Engel JD, Bresnick EH, Zhou J, Shi L. Long non-coding RNA-dependent mechanism to regulate heme biosynthesis and erythrocyte development. Nat Commun 2018 PubMed ID: 30349036 DOI: 10.1038/s41467-018-06883-x

4974 Morrison TA, Wilcox I, Luo HY, Farrell JJ, Kurita R, Nakamura Y, Murphy GJ, Cui S, Steinberg MH, Chui DHK. A long noncoding RNA from the HBS1L-MYB intergenic region on chr6q23 regulates human fetal hemoglobin expression. Blood Cells Mol Dis 2018 69:1-9 PubMed ID: 29227829 DOI: 10.1016/j.bcmd.2017.11.003

6728 Ghosh A, Garee G, Sweeny EA, Nakamura Y, Stuehr DJ. Hsp90 chaperones hemoglobin maturation in erythroid and nonerythroid cells. Proc Natl Acad Sci U S A 2018 115(6):E1117-E1126 PubMed ID: 29358373 DOI: 10.1073/pnas.1717993115

6761 Lee E, Sivalingam J, Lim ZR, Chia G, Shi LG, Roberts M, Loh YH, Reuveny S, Oh SK. Review: In vitro generation of red blood cells for transfusion medicine: Progress, prospects and challenges. Biotechnol Adv 2018 36(8):2118-2128 PubMed ID: 30273713 DOI: 10.1016/j.biotechadv.2018.09.006

6773 Martyn GE, Wienert B, Yang L, Shah M, Norton LJ, Burdach J, Kurita R, Nakamura Y, Pearson RCM, Funnell APW, Quinlan KGR, Crossley M Natural regulatory mutations elevate the fetal globin gene via disruption of BCL11A or ZBTB7A binding. Nat Genet 2018 50(4):498-503 PubMed ID: 29610478 DOI: 10.1038/s41588-018-0085-0

6784 Yu L, Jearawiriyapaisarn N, Lee MP, Hosoya T, Wu Q, Myers G, Lim KC, Kurita R, Nakamura Y, Vojtek AB, Rual JF, Engel JD. BAP1 regulation of the key adaptor protein NCoR1 is critical for γ-globin gene repression. Genes Dev 2018 32(23-24):1537-1549 PubMed ID: 30463901 DOI: 10.1101/gad.318436.118

6785 Sun S, Peng Y, Liu J. Research advances in erythrocyte regeneration sources and methods in vitro. Cell Regen (Lond) 2018 7(2):45-49 PubMed ID: 30671230 DOI: 10.1016/j.cr.2018.10.001

6786 Loucari CC, Patsali P, van Dijk TB, Stephanou C, Papasavva P, Zanti M, Kurita R, Nakamura Y, Christou S, Sitarou M, Philipsen S, Lederer CW, Kleanthous M. Rapid and Sensitive Assessment of Globin Chains for Gene and Cell Therapy of Hemoglobinopathies. Hum Gene Ther Methods 2018 29(1):60-74 PubMed ID: 29325430 DOI: 10.1089/hgtb.2017.190

6787 Vinjamur DS, Bauer DE, Orkin SH Recent progress in understanding and manipulating haemoglobin switching for the haemoglobinopathies. Br J Haematol 2018 180(5):630-643 PubMed ID: 29193029 DOI: 10.1111/bjh.15038

6788 Liu N, Hargreaves VV, Zhu Q, Kurland JV, Hong J, Kim W, Sher F, Macias-Trevino C, Rogers JM, Kurita R, Nakamura Y, Yuan GC, Bauer DE, Xu J, Bulyk ML, Orkin SH. Direct Promoter Repression by BCL11A Controls the Fetal to Adult Hemoglobin Switch. Cell 2018 173(2):430-442.e17 PubMed ID: 29606353 DOI: 10.1016/j.cell.2018.03.016

6790 Khalil S, Delehanty L, Grado S, Holy M, White Z 3rd, Freeman K, Kurita R, Nakamura Y, Bullock G, Goldfarb A. Iron modulation of erythropoiesis is associated with Scribble-mediated control of the erythropoietin receptor. J Exp Med 2018 215(2):661-679 PubMed ID: 29282252 DOI: 10.1084/jem.20170396

6798 Lei Yu, Natee Jearawiriyapaisarn, Mary P. Lee, Tomonori Hosoya, Qingqing Wu, Greggory Myers, Kim-Chew Lim, Ryo Kurita, Yukio Nakamura, Anne B. Vojtek, View ORCID ProfileJean-François Rual, View ORCID ProfileJames Douglas Engel BAP1 deubiquitinase is a potent repressor of fetal hemoglobin biosynthesis bioRxiv 2018 DOI: 10.1101/346619

6807 Shukla A, Huangfu D. Decoding the noncoding genome via large-scale CRISPR screens. Curr Opin Genet Dev 2018 52:70-76 PubMed ID: 29913329 DOI: 10.1016/j.gde.2018.06.001

6813 Wienert B, Martyn GE, Funnell APW, Quinlan KGR, Crossley M. Wake-up Sleepy Gene: Reactivating Fetal Globin for β-Hemoglobinopathies. Trends Genet 2018 34(12):927-940 PubMed ID: 30287096 DOI: 10.1016/j.tig.2018.09.004

6817 Lau CH, Suh Y. CRISPR-based strategies for studying regulatory elements and chromatin structure in mammalian gene control. Mamm Genome 2018 29(3-4):205-228 PubMed ID: 29196861 DOI: 10.1007/s00335-017-9727-2

6819 Paikari A, Sheehan VA. Fetal haemoglobin induction in sickle cell disease. Br J Haematol 2018 180(2):189-200 PubMed ID: 29143315 DOI: 10.1111/bjh.15021

8954 Tyler A Couch, BS , Zachary C. Murphy, PhD , Michael Getman, MS , Ryo Kurita, PhD , Yukio Nakamura, MD PhD , Laurie A. Steiner, MD Human Erythroblasts with c-Kit Activating Mutations Have Reduced Cell Culture Costs and Remain Capable of Terminal Maturation and Enucleation Blood 2018 132 (Supplement 1): 2315 DOI: 10.1182/blood-2018-99-117157

8956 Laura Breda, PhD , Valentina Ghiaccio, PhD , Hanyia Zaidi , Silvia Pires Lourenco, MSc , Carla Casu , Kim Smith-Whitley, MD , Janet L. Kwiatkowski, MD , Vanessa Carrion, RN, CPN , Virginia Guzikowski, RN, MSN, CRNP , Ryo Kurita, PhD , Yukio Nakamura, MD PhD , Improved Lentiviral Vectors for the Cure of Hemoglobinopathies Blood 2018 132 (Supplement 1): 3477 DOI: 10.1182/blood-2018-99-111319

8958 Yuxuan Wu, PhD , Mitchel A. Cole, BS , Abdou Mousas , Jing Zeng, MD , Qiuming Yao, PhD , Divya Vinjamur, PhD , Ryo Kurita, PhD , Yukio Nakamura, MD PhD , Luca Pinello, PhD , Guillame Lettre, PhD , Daniel E. Bauer, MD PhD Comprehensive Integrated Genomic Perturbations Reveal Molecular Mechanisms of Red Blood Cell Trait Associations Blood 2018 132 (Supplement 1): 532 DOI: 10.1182/blood-2018-99-120229

8965 Chiara Antoniani, Vasco Meneghini, Annalisa Lattanzi, Tristan Felix, Oriana Romano, Elisa Magrin, Leslie Weber, Giulia Pavani, Sara El Hoss, Ryo Kurita, Yukio Nakamura, Thomas J Cradick, Ante S Lundberg, Matthew Porteus, Mario Amendola, Wassim El Nemer, Marina Cavazzana, Fulvio Mavilio, Annarita Miccio Induction of Fetal Hemoglobin Synthesis by CRISPR/Cas9-mediated Editing of the Human β-Globin Locus Blood 2018 131 (17), 1960-1973 PubMed ID: 29519807 DOI: 10.1182/blood-2017-10-811505

8968 Tyler A Couch, BS , Zachary C. Murphy, PhD , Jacquelyn Lillis, MS , Michael Getman, MS , Paul D. Kingsley, PhD , Ryo Kurita, PhD , Yukio Nakamura, MD PhD , Laurie A. Steiner, MD High-Level Setd8 Expression Is Important for Establishment of Normal Patterns of Erythroid Gene Expression and Is Essential for Stress Erythropoiesis Blood 2018 132 (Supplement 1): 499 DOI: 10.1182/blood-2018-99-116609

18408 Falak Sher, Vivien Schoonenberg, Mitchel A. Cole, Claudio Macias-Trevino, Paolo Cifani, Patrick G Schupp, Matthew C. Canver, Ryo Kurita, Yukio Nakamur, Stuart Orkin, Alex Kentsi, Luca Pinello, Takahiro Maeda, Daniel E. Bauer High-Resolution CRISPR Functional Genomics Reveals Critical Vulnerabilities of the NuRD Complex for Fetal Hemoglobin Control Blood 2017 130:286 DOI: 10.1182/blood.V130.Suppl_1.286.286

18534 Lu Q, Livi GP, Modha S, Yusa K, Macarrón R, Dow DJ. Applications of CRISPR genome editing technology in drug target identification and validation Expert Opin Drug Discov 2017 12(6):541-552 PubMed ID: 28388235 DOI: 10.1080/17460441.2017.1317244

4973 Wienert B, Martyn GE, Kurita R, Nakamura Y, Quinlan KGR1, Crossley M. KLF1 drives the expression of fetal hemoglobin in British HPFH. Blood 2017 130(6):803-807 PubMed ID: 28659276 DOI: 10.1182/blood-2017-02-767400

6750 Trakarnsanga K, Griffiths RE, Wilson MC, Blair A, Satchwell TJ, Meinders M, Cogan N, Kupzig S, Kurita R, Nakamura Y, Toye AM, Anstee DJ, Frayne J. An immortalized adult human erythroid line facilitates sustainable and scalable generation of functional red cells. Nat Commun 2017 8:14750 PubMed ID: 28290447 DOI: 10.1038/ncomms14750

6765 Yankai Zhang, PhD , Ryo Kurita, PhD , Yukio Nakamura, MD PhD , Vivien Sheehan, MD PhD Screening Fetal Hemoglobin Inducing Compounds in Human Umbilical Cord Blood-Derived Erythroid Progenitor Cells Blood 2017 130 (Supplement 1): 2222 DOI: 10.1182/blood.V130.Suppl_1.2222.2222

6777 Lessard S, Gatof ES, Beaudoin M, Schupp PG, Sher F, Ali A, Prehar S, Kurita R, Nakamura Y, Baena E, Ledoux J, Oceandy D, Bauer DE, Lettre G. An erythroid-specific ATP2B4 enhancer mediates red blood cell hydration and malaria susceptibility. J Clin Invest 2017 127(8):3065-3074 PubMed ID: 28714864 DOI: 10.1172/JCI94378

6778 Canver MC, Lessard S, Pinello L, Wu Y, Ilboudo Y, Stern EN, Needleman AJ, Galactéros F, Brugnara C, Kutlar A, McKenzie C, Reid M, Chen DD, Das PP, A Cole M, Zeng J, Kurita R, Nakamura Y, Yuan GC, Lettre G, Bauer DE, Orkin SH. Variant-aware saturating mutagenesis using multiple Cas9 nucleases identifies regulatory elements at trait-associated loci. Nat Genet 2017 49(4):625-634 PubMed ID: 28218758 DOI: 10.1038/ng.3793

6791 Hodonsky CJ, Jain D, Schick UM, Morrison JV, Brown L, McHugh CP, Schurmann C, Chen DD, Liu YM, Auer PL, Laurie CA, Taylor KD, Browning BL, Li Y, Papanicolaou G1, Rotter JI, Kurita R, Nakamura Y, Browning SR, Loos RJF, North KE, Laurie CC, Thornton TA, Pankratz N, Bauer DE, Sofer T, Reiner AP. Genome-wide association study of red blood cell traits in Hispanics/Latinos: The Hispanic Community Health Study/Study of Latinos. PLoS Genet 2017 13(4):e1006760 PubMed ID: 28453575 DOI: 10.1371/journal.pgen.1006760

6803 Habara AH, Shaikho EM, Steinberg MH. Fetal hemoglobin in sickle cell anemia: The Arab-Indian haplotype and new therapeutic agents. Am J Hematol 2017 92(11):1233-1242 PubMed ID: 28736939 DOI: 10.1002/ajh.24872

6818 Ipe J, Swart M, Burgess KS, Skaar TC. High-Throughput Assays to Assess the Functional Impact of Genetic Variants: A Road Towards Genomic-Driven Medicine. Clin Transl Sci 2017 10(2):67-77 PubMed ID: 28213901 DOI: 10.1111/cts.12440

8824 Yan Dai, PhD , Tyngwei Chen , Heba Ijaz , Elizabeth Cho , Ryo Kurita, PhD , Yukio Nakamura, MD PhD , Martin H. Steinberg, MD SIRT1 Increases the Expression of Fetal Hemoglobin Genes in Human Adult and Cord Blood Erythroblasts Blood 2017 130 (Supplement 1): 287 DOI: 10.1182/blood.V130.Suppl_1.287.287

8955 Silvia Pires Lourenco, MSc , Amaliris Gonzalez, PhD , Tobias Raabe, PhD , Ryo Kurita, PhD , Yukio Nakamura, MD PhD , Laura Breda, PhD , Stefano Rivella, PhD Generation of New Cell Models to Screen Improved Vectors for Functional Hemoglobin Expression Blood 2017 130 (Supplement 1): 5563 DOI: 10.1182/blood.V130.Suppl_1.5563.5563

8962 Laura J Norton, Alister P W Funnell, Jon Burdach, Beeke Wienert, Ryo Kurita, Yukio Nakamura, Sjaak Philipsen, Richard C M Pearson, Kate G R Quinlan, Merlin Crossley KLF1 Directly Activates Expression of the Novel Fetal Globin Repressor ZBTB7A/LRF in Erythroid Cells Blood Adv 2017 1 (11), 685-692 PubMed ID: 29296711 DOI: 10.1182/bloodadvances.2016002303

8966 Shadi Khalil, Maja Holy, Stephen Grado, Robert Fleming, Ryo Kurita, Yukio Nakamura, Adam Goldfarb A Specialized Pathway for Erythroid Iron Delivery Through Lysosomal Trafficking of Transferrin Receptor 2 Blood Adv 2017 1 (15), 1181-1194 PubMed ID: 29296759 DOI: 10.1182/bloodadvances.2016003772

8967 Brian M Dulmovits, MS , Julien Papoin, MS , Jian Hua Li, MS , Ryo Kurita, PhD , Yukio Nakamura, MD PhD , Yousef Al-Abed, MD , Luanne L. Peters, PhD , Betty Diamond, MD , Anupama Narla, MD , Jeffrey M Lipton, MDPhD , Mohandas Narla, DSc , Kevin Tracey, MD , Lionel Blanc, PhD High Mobility Group Box 1 Protein (HMGB1) Mediates Anemia of Inflammation through Lineage Skewing and Direct Inhibition of EPO Signaling Blood 2017 130 (Supplement 1): 2196 DOI: 10.1182/blood.V130.Suppl_1.2196.2196

18518 Hu X. CRISPR/Cas9 system and its applications in human hematopoietic cells Blood Cells Mol Dis 2016 62:6-12 PubMed ID: 27736664 DOI: 10.1016/j.bcmd.2016.09.003

18530 Sripichai O, Fucharoen S. Fetal hemoglobin regulation in β-thalassemia: heterogeneity, modifiers and therapeutic approaches Expert Rev Hematol 2016 9(12):1129-1137. PubMed ID: 27801605 DOI: 10.1080/17474086.2016.1255142

6762 Masuda T, Wang X, Maeda M, Canver MC, Sher F, Funnell AP, Fisher C, Suciu M, Martyn GE, Norton LJ, Zhu C, Kurita R, Nakamura Y, Xu J, Higgs DR, Crossley M, Bauer DE, Orkin SH, Kharchenko PV, Maeda T. Transcription factors LRF and BCL11A independently repress expression of fetal hemoglobin. Science 2016 351(6270):285-9 PubMed ID: 26816381 DOI: 10.1126/science.aad3312

6764 Traxler EA, Yao Y, Wang YD, Woodard KJ, Kurita R, Nakamura Y, Hughes JR, Hardison RC, Blobel GA, Li C, Weiss MJ. A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition. Nat Med 2016 22(9):987-90 PubMed ID: 27525524 DOI: 10.1038/nm.4170

6774 Orkin SH. Recent advances in globin research using genome-wide association studies and gene editing. Ann N Y Acad Sci 2016 368(1):5-10 PubMed ID: 26866328 DOI: 10.1111/nyas.13001

6795 Smith EC, Orkin SH. Hemoglobin genetics: recent contributions of GWAS and gene editing. Hum Mol Genet 2016 25(R2):R99-R105 PubMed ID: 27340226 DOI: 10.1093/hmg/ddw170

6802 Wright JB, Sanjana NE. CRISPR Screens to Discover Functional Noncoding Elements. Trends Genet 2016 32(9):526-529 PubMed ID: 27423542 DOI: 10.1016/j.tig.2016.06.004

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8963 Sriram Krishnamoorthy , Dipti Gupta , William E. Hobbs, MD PhD , Christine Loh, PhD , David R. Light , Robert Peters, PhD , Sarah Sturtevant , Betty Pace, MD , Yukio Nakamura , Brian Lucas , Benjamin F. Vieira Nrf2 activation and fetal hemoglobin induction in sickle cell disease Blood 2016 128 (22): 4840 DOI: 10.1182/blood.V128.22.4840.4840

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22109 Lv A, Chen M, Zhang S, Zhao W, Li J, Lin S, Zheng Y, Lin N, Xu L, Huang H. Upregulation of miR‑6747‑3p affects red blood cell lineage development and induces fetal hemoglobin expression by targeting BCL11A in β‑thalassemia. Mol Med Rep 2025 31(1) PubMed ID: 39450557 DOI: 10.3892/mmr.2024.13372

22556 Yang X, Cheng L, Xin Y, Zhang J, Chen X, Xu J, Zhang M, Feng R, Hyle J, Qi W, Rosikiewicz W, Xu B, Li C, Xu P. CTCF is selectively required for maintaining chromatin accessibility and gene expression in human erythropoiesis. Genome Biol 2025 26(1):44 PubMed ID: 40022213 DOI: 10.1186/s13059-025-03510-z

22577 Ping J, Liu X, Lu Y, Quan C, Fan P, Lu H, Li Q, Wang C, Zhang Z, Liu M, Chen S, Chang L, Jiang Y, Huang Q, Liu J, Wuren T, Liu H, Hao Y, Kang L, Liu G, Lu H, Wei X, Wang Y, Li Y, Guo H, Cui Y, Zhang H, Zhang Y, Zhai Y, He Y, Zheng W, Qi X, Ouzhuluobu, Ma H, Yang L, Wang X, Jin W, Cui Y, Ge R, Wu S, Wei Y, Su B, He F, Zhang H, Zhou G. A highland-adaptation variant near MCUR1 reduces its transcription and attenuates erythrogenesis in Tibetans. Cell Genom 2025 100782 PubMed ID: 40043709 DOI: 10.1016/j.xgen.2025.100782

22758 Setegne MT, Cabral AT, Tiwari A, Shen F, Thiam HR, Dassama LMK. Engineering Cell-Specific Protein Delivery Vehicles for Erythroid Lineage Cells. ACS Bio Med Chem Au 2025 5(2):268-282 PubMed ID: 40255284 DOI: 10.1021/acsbiomedchemau.4c00098

23019 Miyazawa M, Kawai K, Yamamoto S, Kitagawa D. Protocol for immunostaining of non-adherent cells and cellular structures using centrifugal filter devices. STAR Protoc 2025 6(3):103934 PubMed ID: 40616839 DOI: 10.1016/j.xpro.2025.103934

22286 Li J, Chen M, Zhao W, Lv A, Lin S, Zheng Y, Cai M, Lin N, Xu L, Huang H. The role of miR-129-5p in regulating γ-globin expression and erythropoiesis in β-thalassemia. Hum Mol Genet 2024 PubMed ID: 39657657 DOI: 10.1093/hmg/ddae180

22295 Yang JP, Toughiri R, Gounder AP, Scheibe D, Petrus M, Fink SJ, Vallee S, Kenniston J, Papaioannou N, Langston S, Gavva NR, Horman SR. Identification of small molecule agonists of fetal hemoglobin expression for the treatment of sickle cell disease PLoS One 2024 19(11):e0307049 PubMed ID: 39504332 DOI: 10.1371/journal.pone.0307049

22857 Shao C, Liu Q, Xu J, Zhang J, Zhang C, Xin Y, Ye Y, Lin B, Zhang X, Cheng L, Xu X, Xu P. Efficient and in situ correction of hemoglobin Constant Spring mutation by prime editing in human hematopoietic cells Mol Ther Nucleic Acids 2024 35(4):102371 PubMed ID: 39640014 DOI: 10.1016/j.omtn.2024.102371

22858 Han X, He W, Liang D, Liu X, Zhou J, de Thé H, Zhu J, Yuan H. Creg1 Regulates Erythroid Development via TGF-β/Smad2-Klf1 Axis in Zebrafish Adv Sci (Weinh) 2024 11(33):e2402804 PubMed ID: 38953462 DOI: 10.1002/advs.202402804

22913 Martyn GE, Doerfler PA, Yao Y, Quinlan KGR, Weiss MJ, Crossley M. Hydroxyurea reduces the levels of the fetal globin gene repressors ZBTB7A/LRF and BCL11A in erythroid cells in vitro J Sick Cell Dis 2024 1(1):yoae008. PubMed ID: 40304012 DOI: 10.1093/jscdis/yoae008

17723 Mehta S, Buyanbat A, Orkin S, Nabet B. High-efficiency knock-in of degradable tags (dTAG) at endogenous loci in cell lines Methods Enzymol 2023 681:1-22 PubMed ID: 36764753 DOI: 10.1016/bs.mie.2022.08.045

18374 Wu J, Moriwaki K, Asuka T, Nakai R, Kanda S, Taniguchi M, Sugiyama T, Yoshimura SI, Kunii M, Nagasawa T, Hosen N, Miyoshi E, Harada A. EHBP1L1, an apicobasal polarity regulator, is critical for nuclear polarization during enucleation of erythroblasts Blood Adv 2023 7(14):3382-3394 PubMed ID: 37042948 DOI: 10.1182/bloodadvances.2022008930

18393 Zhang Y, Xie H, Liang G, Qin Y, Wei X, Ning S, Liang Y, Liang X, Xie Y, Lin Z, Zhu D, Lin J, Xiong F, Xu X, Shang X. A novel gain-of-function PIP4K2A mutation elevates the expression of β-globin and aggravates the severity of α-thalassemia Br J Haematol 2023 202(5):1018-1023 PubMed ID: 37423903 DOI: 10.1111/bjh.18967

18399 Boontanrart MY, Mächler E, Ponta S, Nelis JC, Preiano VG, Corn JE. Engineering of the endogenous HBD promoter increases HbA2 Elife 2023 12:e85258 PubMed ID: 37265399 DOI: 10.7554/eLife.85258

18402 Shang S, Li X, Azzo A, Truong T, Dozmorov M, Lyons C, Manna AK, Williams DC Jr, Ginder GD. MBD2a-NuRD binds to the methylated γ-globin gene promoter and uniquely forms a complex required for silencing of HbF expression Proc Natl Acad Sci U S A 2023 120(25):e2302254120 PubMed ID: 37307480 DOI: 10.1073/pnas.2302254120

18434 Yang F, Xu S, Huang C, Shao Z, Hu Y, Yang Yang, Lai Y, Ke Q, Liang D, Wu Y. Therapeutic genome editing of an aberrant splice site in β-thalassemia by CRISPR/Cas9 with multiple sgRNAs Genes Dis 2023 11(1):15-18 PubMed ID: 37588192 DOI: 10.1016/j.gendis.2023.01.010

18436 Leighton GO, Shang S, Hageman S, Ginder GD, Williams DC Jr. Analysis of the complex between MBD2 and the histone deacetylase core of NuRD reveals key interactions critical for gene silencing Proc Natl Acad Sci U S A 2023 120(33):e2307287120 PubMed ID: 37552759 DOI: 10.1073/pnas.2307287120

18437 Wang X, McKillop WM, Dlugi TA, Faber ML, Alvarez-Argote J, Chambers CB, Wilber A, Medin JA. A mass spectrometry assay for detection of endogenous and lentiviral engineered hemoglobin in cultured cells and sickle cell disease mice J Gene Med 2023 e3567 PubMed ID: 37455676 DOI: 10.1002/jgm.3567

18438 Xue N, Liu X, Zhang D, Wu Y, Zhong Y, Wang J, Fan W, Jiang H, Zhu B, Ge X, Gonzalez RVL, Chen L, Zhang S, She P, Zhong Z, Sun J, Chen X, Wang L, Gu Z, Zhu P, Liu M, Li D, Zhong TP, Zhang X. Improving adenine and dual base editors through introduction of TadA-8e and Rad51DBD Nat Commun 2023 14(1):1224 PubMed ID: 36869044 DOI: 10.1038/s41467-023-36887-1

18444 Baro B, Kim CY, Lin C, Kongsomboonvech AK, Tetard M, Peterson NA, Salinas ND, Tolia NH, Egan ES. Plasmodium falciparum exploits CD44 as a co-receptor for erythrocyte invasion Blood 2023 2023020831 PubMed ID: 37832027 DOI: 10.1182/blood.2023020831

18447 Cervellera CF, Mazziotta C, Di Mauro G, Iaquinta MR, Mazzoni E, Torreggiani E, Tognon M, Martini F, Rotondo JC. Immortalized erythroid cells as a novel frontier for in vitro blood production: current approaches and potential clinical application Stem Cell Res Ther 2023 14(1):139 PubMed ID: 37226267 DOI: 10.1186/s13287-023-03367-8

18489 Kaur G, Ren R, Hammel M, Horton JR, Yang J, Cao Y, He C, Lan F, Lan X, Blobel GA, Blumenthal RM, Zhang X, Cheng X. Allosteric autoregulation of DNA binding via a DNA-mimicking protein domain: a biophysical study of ZNF410-DNA interaction using small angle X-ray scattering Nucleic Acids Res 2023 51(4):1674-1686 PubMed ID: 36660822 DOI: 10.1093/nar/gkac1274

18499 Ren R, Horton JR, Chen Q, Yang J, Liu B, Huang Y, Blumenthal RM, Zhang X, Cheng X. Structural basis for transcription factor ZBTB7A recognition of DNA and effects of ZBTB7A somatic mutations that occur in human acute myeloid leukemia J Biol Chem 2023 299(2):102885 PubMed ID: 36626981 DOI: 10.1016/j.jbc.2023.102885

18502 Nai A, Cordero-Sanchez C, Tanzi E, Pagani A, Silvestri L, Di Modica SM. Cellular and animal models for the investigation of β-thalassemia Blood Cells Mol Dis 2023 102761 PubMed ID: 37271682 DOI: 10.1016/j.bcmd.2023.102761

18506 Zhou Y, Dogiparthi VR, Ray S, Schaefer MA, Harris HL, Rowley MJ, Hewitt KJ. Defining a cohort of anemia-activated cis elements reveals a mechanism promoting erythroid precursor function Blood Adv 2023 7(20):6325-6338 PubMed ID: 36809789 DOI: 10.1182/bloodadvances.2022009163

18511 Brusson M, Chalumeau A, Martinucci P, Romano O, Felix T, Poletti V, Scaramuzza S, Ramadier S, Masson C, Ferrari G, Mavilio F, Cavazzana M, Amendola M, Miccio A. Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies Mol Ther Nucleic Acids 2023 32:229-246 PubMed ID: 37090420 DOI: 10.1016/j.omtn.2023.03.012

21007 Yin M, Izadi M, Tenglin K, Viennet T, Zhai L, Zheng G, Arthanari H, Dassama LMK, Orkin SH. Evolution of nanobodies specific for BCL11A. Proc Natl Acad Sci U S A 2023 120(3) PubMed ID: 36626555 DOI: 10.1073/pnas.2218959120

21187 Badat M, Ejaz A, Hua P, Rice S, Zhang W, Hentges LD, Fisher CA, Denny N, Schwessinger R, Yasara N, Roy NBA, Issa F, Roy A, Telfer P, Hughes J, Mettananda S, Higgs DR, Davies JOJ. Direct correction of haemoglobin E β-thalassaemia using base editors. Nat Commun 2023 14(1):2238 PubMed ID: 37076455 DOI: 10.1038/s41467-023-37604-8

21274 Chaand M, Fiore C, Johnston B, D'Ippolito A, Moon DH, Carulli JP, Shearstone JR. Erythroid lineage chromatin accessibility maps facilitate identification and validation of NFIX as a fetal hemoglobin repressor. Commun Biol 2023 6(1):640 PubMed ID: 37316562 DOI: 10.1038/s42003-023-05025-4

21304 Mayuranathan T, Newby GA, Feng R, Yao Y, Mayberry KD, Lazzarotto CR, Li Y, Levine RM, Nimmagadda N, Dempsey E, Kang G, Porter SN, Doerfler PA, Zhang J, Jang Y, Chen J, Bell HW, Crossley M, Bhoopalan SV, Sharma A, Tisdale JF, Pruett-Miller SM, Cheng Y, Tsai SQ, Liu DR, Weiss MJ, Yen JS. Potent and uniform fetal hemoglobin induction via base editing. Nat Genet 2023 PubMed ID: 37400614 DOI: 10.1038/s41588-023-01434-7

21452 Simbula M, Manchinu MF, Mingoia M, Pala M, Asunis I, Caria CA, Perseu L, Shah M, Crossley M, Moi P, Ristaldi MS. miR-365-3p mediates BCL11A and SOX6 erythroid-specific coregulation: A new player in HbF activation. Mol Ther Nucleic Acids 2023 34:102025 PubMed ID: 37744176 DOI: 10.1016/j.omtn.2023.09.002

21464 Ravi NS, George A, Mohankumar KM. Protocol for arrayed gRNA screening by base editors in mammalian cell lines using lentiviral system. STAR Protoc 2023 4(4):102668 PubMed ID: 37922314 DOI: 10.1016/j.xpro.2023.102668

21484 Han W, Qiu HY, Sun S, Fu ZC, Wang GQ, Qian X, Wang L, Zhai X, Wei J, Wang Y, Guo YL, Cao GH, Ji RJ, Zhang YZ, Ma H, Wang H, Zhao M, Wu J, Bi L, Chen QB, Li Z, Yu L, Mou X, Yin H, Yang L, Chen J, Yang B, Zhang Y. Base editing of the HBG promoter induces potent fetal hemoglobin expression with no detectable off-target mutations in human HSCs. Cell Stem Cell 2023 PubMed ID: 37989316 DOI: 10.1016/j.stem.2023.10.007

17576 Forester CM, Oses-Prieto JA, Phillips NJ, Miglani S, Pang X, Byeon GW, DeMarco R, Burlingame A, Barna M, Ruggero D. Regulation of eIF4E guides a unique translational program to control erythroid maturation Sci Adv 2022 8(51):eadd3942 PubMed ID: 36563140 DOI: 10.1126/sciadv.add3942

17720 Wang D, Tanaka-Yano M, Meader E, Kinney MA, Morris V, Lummertz da Rocha E, Liu N, Liu T, Zhu Q, Orkin SH, North TE, Daley GQ, Rowe RG. Developmental maturation of the hematopoietic system controlled by a Lin28b-let-7-Cbx2 axis Cell Rep 2022 39(1):110587 PubMed ID: 35385744 DOI: 10.1016/j.celrep.2022.110587

18195 Park SH, Cao M, Pan Y, Davis TH, Saxena L, Deshmukh H, Fu Y, Treangen T, Sheehan VA, Bao G. Comprehensive analysis and accurate quantification of unintended large gene modifications induced by CRISPR-Cas9 gene editing Sci Adv 2022 8(42):eabo7676 PubMed ID: 36269834 DOI: 10.1126/sciadv.abo7676

18371 Topfer SK, Feng R, Huang P, Ly LC, Martyn GE, Blobel GA, Weiss MJ, Quinlan KGR, Crossley M. Disrupting the adult globin promoter alleviates promoter competition and reactivates fetal globin gene expression Blood 2022 139(14):2107-2118 PubMed ID: 35090172 DOI: 10.1182/blood.2021014205

18373 Shrestha D, Bag A, Wu R, Zhang Y, Tang X, Qi Q, Xing J, Cheng Y. Genomics and epigenetics guided identification of tissue-specific genomic safe harbors Genome Biol 2022 23(1):199 PubMed ID: 36131352 DOI: 10.1186/s13059-022-02770-3

18382 Zhang H, Zhou Q, Chen H, Lu D. Prime Editor 3 Mediated Beta-Thalassemia Mutations of the HBB Gene in Human Erythroid Progenitor Cells Int J Mol Sci 2022 23(9):5002 PubMed ID: 35563395 DOI: 10.3390/ijms23095002

18390 Ginette Balbin-Cuesta, Katherine Rybkin, Greggory Myers, Rami Khoriaty Identifying Novel Regulators of Erythroid Maturation and Proliferation Using a Genome-Scale CRISPR Activation Screen Blood 2022 140:8170-8171 DOI: 10.1182/blood-2022-170108

18391 Yannis Hara, Emily Kawabata, Viktor T. Lemgart, Paola G. Bronson, Alexandra Hicks, Sriram Krishnamoorthy, Nicholas Watkins, Robert Peters, David J. Roberts, Emanuele Di Angelantonio, John Danesh, William Astle, Dirk S. Paul, Samuel Lessard, Adam S. Butterworth Inhibition of MRP1 Induces Fetal Hemoglobin through NRF2 Activation to Protect Human Erythroid Cells from Sickling Blood 2022 140:952-953 DOI: 10.1182/blood-2022-168169

18396 Greggory Myers, Lei Yu, Ginette Balbin-Cuesta, Ayse Bilge Ozel, James Douglas Engel, Rami Khoriaty Identification of Novel Regulators of Erythropoiesis Using Whole-Genome CRISPR-Cas9 Screening Blood 2022 140:8155-8156 DOI: 10.1182/blood-2022-170101

18409 Charlene Lam, Kevin Gillinder, Natalia Carvajal, Helen Lorraine Mitchell, Graham William Magor, Andrew Charles Perkins Novel Immediate Direct Targets of EPO Signalling in Human Erythropoiesis Blood 2022 140:1238-1238 DOI: 10.1182/blood-2022-168671

18410 David C. Williams, Gage Oliver Leighton, Parnika Agrawal, Glory Dan-Dukor, Shengzhe Shang, Gordon Ginder ETO2 Recruitment of NuRD to the γ-Globin Locus Involves Multivalent Interaction with GATAD2A Blood 2022 140:1939-1940 DOI: 10.1182/blood-2022-169694

18428 Shen F, Zheng G, Setegne M, Tenglin K, Izadi M, Xie H, Zhai L, Orkin SH, Dassama LMK. A Cell-Permeant Nanobody-Based Degrader That Induces Fetal Hemoglobin ACS Cent Sci 2022 8(12):1695-1703 PubMed ID: 36589886 DOI: 10.1021/acscentsci.2c00998

18435 Sun Y, Habara A, Le CQ, Nguyen N, Chen R, Murphy GJ, Chui DHK, Steinberg MH, Cui S. Pharmacologic induction of PGC-1α stimulates fetal haemoglobin gene expression Br J Haematol 2022 197(1):97-109 PubMed ID: 35118652 DOI: 10.1111/bjh.18042

18443 Mehta S, Buyanbat A, Kai Y, Karayel O, Goldman SR, Seruggia D, Zhang K, Fujiwara Y, Donovan KA, Zhu Q, Yang H, Nabet B, Gray NS, Mann M, Fischer ES, Adelman K, Orkin SH. Temporal resolution of gene derepression and proteome changes upon PROTAC-mediated degradation of BCL11A protein in erythroid cells Cell Chem Biol 2022 29(8):1273-1287 PubMed ID: 35839780 DOI: 10.1016/j.chembiol.2022.06.007

18492 Ramadier S, Chalumeau A, Felix T, Othman N, Aknoun S, Casini A, Maule G, Masson C, De Cian A, Frati G, Brusson M, Concordet JP, Cavazzana M, Cereseto A, El Nemer W, Amendola M, Wattellier B, Meneghini V, Miccio A. Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease Mol Ther 2022 30(1):145-163 PubMed ID: 34418541 DOI: 10.1016/j.ymthe.2021.08.019

18500 Yichao Zhou, Linda Chee, Suhita Ray, Meg Schaefer, Venkatasai Rahul Dogiparthi, Jordan Rowley, Kyle J Hewitt Anemia-Activated Cis Regulatory Requirements for Erythroid Regeneration Blood 2022 140:701-701 DOI: 10.1182/blood-2022-167544

18509 Steinberg MH. Targeting fetal hemoglobin expression to treat β hemoglobinopathies Expert Opin Ther Targets 2022 26(4):347-359 PubMed ID: 35418266 DOI: 10.1080/14728222.2022.2066519

18564 Bircher C, Bieri J, Assaraf R, Leisi R, Ros C. A Conserved Receptor-Binding Domain in the VP1u of Primate Erythroparvoviruses Determines the Marked Tropism for Erythroid Cells Viruses 2022 14(2):420 PubMed ID: 35216013 DOI: 10.3390/v14020420

20716 Qin K, Huang P, Feng R, Keller CA, Peslak SA, Khandros E, Saari MS, Lan X, Mayuranathan T, Doerfler PA, Abdulmalik O, Giardine B, Chou ST, Shi J, Hardison RC, Weiss MJ, Blobel GA. Dual function NFI factors control fetal hemoglobin silencing in adult erythroid cells. Nat Genet 2022 PubMed ID: 35618846 DOI: 10.1038/s41588-022-01076-1

20782 Patino CA, Pathak N, Mukherjee P, Park SH, Bao G, Espinosa HD. Multiplexed high-throughput localized electroporation workflow with deep learning-based analysis for cell engineering. Sci Adv 2022 8(29):eabn7637 PubMed ID: 35867793 DOI: 10.1126/sciadv.abn7637

20889 Feng R, Mayuranathan T, Huang P, Doerfler PA, Li Y, Yao Y, Zhang J, Palmer LE, Mayberry K, Christakopoulos GE, Xu P, Li C, Cheng Y, Blobel GA, Simon MC, Weiss MJ. Activation of γ-globin expression by hypoxia-inducible factor 1α. Nature 2022 PubMed ID: 36224385 DOI: 10.1038/s41586-022-05312-w

20924 Antoniou P, Hardouin G, Martinucci P, Frati G, Felix T, Chalumeau A, Fontana L, Martin J, Masson C, Brusson M, Maule G, Rosello M, Giovannangeli C, Abramowski V, de Villartay JP, Concordet JP, Del Bene F, El Nemer W, Amendola M, Cavazzana M, Cereseto A, Romano O, Miccio A. Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression. Nat Commun 2022 13(1):6618 PubMed ID: 36333351 DOI: 10.1038/s41467-022-34493-1

18369 Hu Y, Stilp AM, McHugh CP, Rao S, Jain D, Zheng X, Lane J, Méric de Bellefon S, Raffield LM, Chen MH, Yanek LR, Wheeler M, Yao Y, Ren C, Broome J, Moon JY, de Vries PS, Hobbs BD, Sun Q, Surendran P, Brody JA, Blackwell TW, Choquet H, Ryan K, Duggirala R, Heard-Costa N, Wang Z, Chami N, Preuss MH, Min N, Ekunwe L, Lange LA, Cushman M, Faraday N, Curran JE, Almasy L, Kundu K, Smith AV, Gabriel S, Rotter JI, Fornage M, Lloyd-Jones DM, Vasan RS, Smith NL, North KE, Boerwinkle E, Becker LC, Lewis JP, Abecasis GR, Hou L, O'Connell JR, Morrison AC, Beaty TH, Kaplan R, Correa A, Blangero J, Jorgenson E, Psaty BM, Kooperberg C, Walton RT, Kleinstiver BP, Tang H, Loos RJF, Soranzo N, Butterworth AS, Nickerson D, Rich SS, Mitchell BD, Johnson AD, Auer PL, Li Y, Mathias RA, Lettre G, Pankratz N, Laurie CC, Laurie CA, Bauer DE, Conomos MP, Reiner AP, NHLBI Trans-Omics for Precision Medicine (TOPMed) Consortium. Whole-genome sequencing association analysis of quantitative red blood cell phenotypes: The NHLBI TOPMed program Am J Hum Genet 2021 108(5):874-893 PubMed ID: 33887194 DOI: 10.1016/j.ajhg.2021.04.003

18370 Uchida N, Ferrara F, Drysdale CM, Yapundich M, Gamer J, Nassehi T, DiNicola J, Shibata Y, Wielgosz M, Kim YS, Bauler M, Throm RE, Haro-Mora JJ, Demirci S, Bonifacino AC, Krouse AE, Linde NS, Donahue RE, Ryu B, Tisdale JF. Sustained fetal hemoglobin induction in vivo is achieved by BCL11A interference and coexpressed truncated erythropoietin receptor Sci Transl Med 2021 13(591):eabb0411 PubMed ID: 33910976 DOI: 10.1126/scitranslmed.abb0411

18383 Cheng Y, Shang X, Chen D, Pang D, Zhao C, Xu X. MicroRNA-2355-5p regulates γ-globin expression in human erythroid cells by inhibiting KLF6 Br J Haematol 2021 193(2):401-405 PubMed ID: 33368182 DOI: 10.1111/bjh.17134

18384 Ruopeng Feng, Thiyagaraj Mayuranathan, Peng Huang, Phillip A Doerfler, Yichao Li, Yu Yao, Jingjing Zhang, Lance E. Palmer, Peng Xu, Kalin Mayberry, Shaela Wright, Chunliang Li, Yong Cheng, Gerd A. Blobel, M. Celeste Simon, Mitchell J. Weiss Regulation of Fetal Hemoglobin Expression By the VHL-HIF1α Oxygen Sensing System Blood 2021 138:574-574 DOI: 10.1182/blood-2021-149901

18388 Lin J, Ye Y, Shang X, Zhang Y, Wei X, Xu X. TEA domain transcription factor 4 modulates repression of fetal haemoglobin by direct binding to the γ-globin gene promoters Br J Haematol 2021 195(5):764-769 PubMed ID: 34569056 DOI: 10.1111/bjh.17786

18389 Gong Y, Zhang X, Zhang Q, Zhang Y, Ye Y, Yu W, Shao C, Yan T, Huang J, Zhong J, Wang L, Li Y, Wang L, Xu X. A natural DNMT1 mutation elevates the fetal hemoglobin level via epigenetic derepression of the γ-globin gene in β-thalassemia Blood 2021 137(12):1652-1657 PubMed ID: 33227819 DOI: 10.1182/blood.2020006425

18400 Li C, Georgakopoulou A, Mishra A, Gil S, Hawkins RD, Yannaki E, Lieber A. In vivo HSPC gene therapy with base editors allows for efficient reactivation of fetal γ-globin in β-YAC mice Blood Adv 2021 5(4):1122-1135 PubMed ID: 33620431 DOI: 10.1182/bloodadvances.2020003702

18406 Pavani G, Fabiano A, Laurent M, Amor F, Cantelli E, Chalumeau A, Maule G, Tachtsidi A, Concordet JP, Cereseto A, Mavilio F, Ferrari G, Miccio A, Amendola M. Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cells Blood Adv 2021 5(5):1137-1153 PubMed ID: 33635334 DOI: 10.1182/bloodadvances.2020001996

18431 Boddu PC, Gupta AK, Kim JS, Neugebauer KM, Waldman T, Pillai MM. Generation of scalable cancer models by combining AAV-intron-trap, CRISPR/Cas9, and inducible Cre-recombinase Commun Biol 2021 4(1):1184 PubMed ID: 34645977 DOI: 10.1038/s42003-021-02690-1

18439 Lan X, Ren R, Feng R, Ly LC, Lan Y, Zhang Z, Aboreden N, Qin K, Horton JR, Grevet JD, Mayuranathan T, Abdulmalik O, Keller CA, Giardine B, Hardison RC, Crossley M, Weiss MJ, Cheng X, Shi J, Blobel GA. ZNF410 Uniquely Activates the NuRD Component CHD4 to Silence Fetal Hemoglobin Expression Mol Cell 2021 81(2):239-254.e8 PubMed ID: 33301730 DOI: 10.1016/j.molcel.2020.11.006

18440 Nualkaew T, Sii-Felice K, Giorgi M, McColl B, Gouzil J, Glaser A, Voon HPJ, Tee HY, Grigoriadis G, Svasti S, Fucharoen S, Hongeng S, Leboulch P, Payen E, Vadolas J. Coordinated β-globin expression and α2-globin reduction in a multiplex lentiviral gene therapy vector for β-thalassemia Mol Ther 2021 29(9):2841-2853 PubMed ID: 33940155 DOI: 10.1016/j.ymthe.2021.04.037

18442 Shen Y, Verboon JM, Zhang Y, Liu N, Kim YJ, Marglous S, Nandakumar SK, Voit RA, Fiorini C, Ejaz A, Basak A, Orkin SH, Xu J, Sankaran VG. A unified model of human hemoglobin switching through single-cell genome editing Nat Commun 2021 12(1):4991 PubMed ID: 34404810 DOI: 10.1038/s41467-021-25298-9

18446 Li C, Wang H, Georgakopoulou A, Gil S, Yannaki E, Lieber A. In Vivo HSC Gene Therapy Using a Bi-modular HDAd5/35++ Vector Cures Sickle Cell Disease in a Mouse Model Mol Ther 2021 29(2):822-837 PubMed ID: 32949495 DOI: 10.1016/j.ymthe.2020.09.001

18491 King AJ, Songdej D, Downes DJ, Beagrie RA, Liu S, Buckley M, Hua P, Suciu MC, Marieke Oudelaar A, Hanssen LLP, Jeziorska D, Roberts N, Carpenter SJ, Francis H, Telenius J, Olijnik AA, Sharpe JA, Sloane-Stanley J, Eglinton J, Kassouf MT, Orkin SH, Pennacchio LA, Davies JOJ, Hughes JR, Higgs DR, Babbs C. Reactivation of a developmentally silenced embryonic globin gene Nat Commun 2021 12(1):4439 PubMed ID: 34290235 DOI: 10.1038/s41467-021-24402-3

18504 Psatha N, Georgakopoulou A, Li C, Nandakumar V, Georgolopoulos G, Acosta R, Paschoudi K, Nelson J, Chee D, Athanasiadou A, Kouvatsi A, Funnell APW, Lieber A, Yannaki E, Papayannopoulou T. Enhanced HbF reactivation by multiplex mutagenesis of thalassemic CD34+ cells in vitro and in vivo Blood 2021 138(17):1540-1553 PubMed ID: 34086867 DOI: 10.1182/blood.2020010020

20126 Qi Q, Cheng L, Tang X, He Y, Li Y, Yee T, Shrestha D, Feng R, Xu P, Zhou X, Pruett-Miller S, Hardison RC, Weiss MJ, Cheng Y. Dynamic CTCF binding directly mediates interactions among cis-regulatory elements essential for hematopoiesis. Blood 2021 137(10):1327-1339 PubMed ID: 33512425 DOI: 10.1182/blood.2020005780

20255 Doerfler PA, Feng R, Li Y, Palmer LE, Porter SN, Bell HW, Crossley M, Pruett-Miller SM, Cheng Y, Weiss MJ. Activation of γ-globin gene expression by GATA1 and NF-Y in hereditary persistence of fetal hemoglobin. Nat Genet 2021 53(8):1177-1186 PubMed ID: 34341563 DOI: 10.1038/s41588-021-00904-0

20388 Downes DJ, Cross AR, Hua P, Roberts N, Schwessinger R, Cutler AJ, Munis AM, Brown J, Mielczarek O, de Andrea CE, Melero I, COvid-19 Multi-omics Blood ATlas (COMBAT) Consortium, Gill DR, Hyde SC, Knight JC, Todd JA, Sansom SN, Issa F, Davies JOJ, Hughes JR. Identification of LZTFL1 as a candidate effector gene at a COVID-19 risk locus. Nat Genet 2021 53(11):1606-1615 PubMed ID: 34737427 DOI: 10.1038/s41588-021-00955-3

20449 Georgolopoulos G, Psatha N, Iwata M, Nishida A, Som T, Yiangou M, Stamatoyannopoulos JA, Vierstra J. Discrete regulatory modules instruct hematopoietic lineage commitment and differentiation. Nat Commun 2021 12(1):6790 PubMed ID: 34815405 DOI: 10.1038/s41467-021-27159-x

16052 Xu P, Scott DC, Xu B, Yao Y, Feng R, Cheng L, Mayberry K, Wang YD, Bi W, Palmer LE, King MT, Wang H, Li Y, Fan Y, Alpi AF, Li C, Peng J, Papizan J, Pruett-Miller SM, Spallek R, Bassermann F, Cheng Y, Schulman BA, Weiss MJ. FBXO11-mediated proteolysis of BAHD1 relieves PRC2-dependent transcriptional repression in erythropoiesis Blood 2021 137(2):155-167. PubMed ID: 33156908 DOI: 10.1182/blood.2020007809

16055 Liu N, Xu S, Yao Q, Zhu Q, Kai Y, Hsu JY, Sakon P, Pinello L, Yuan GC, Bauer DE, Orkin SH. Transcription factor competition at the γ-globin promoters controls hemoglobin switching Nat Genet 2021 53(4):511-520 PubMed ID: 33649594 DOI: 10.1038/s41588-021-00798-y

16059 Cheng L, Li Y, Qi Q, Xu P, Feng R, Palmer L, Chen J, Wu R, Yee T, Zhang J, Yao Y, Sharma A, Hardison RC, Weiss MJ, Cheng Y. Single-nucleotide-level mapping of DNA regulatory elements that control fetal hemoglobin expression Nat Genet 2021 53(6):869-880 PubMed ID: 33958780 DOI: 10.1038/s41588-021-00861-8

16060 Vinjamur DS, Yao Q, Cole MA, McGuckin C, Ren C, Zeng J, Hossain M, Luk K, Wolfe SA, Pinello L, Bauer DE. ZNF410 represses fetal globin by singular control of CHD4 Nat Genet 2021 53(5):719-728 PubMed ID: 33859416 DOI: 10.1038/s41588-021-00843-w

16062 Demers M, Sturtevant S, Guertin KR, Gupta D, Desai K, Vieira BF, Li W, Hicks A, Ismail A, Gonçalves BP, Di Caprio G, Schonbrun E, Hansen S, Musayev FN, Safo MK, Wood DK, Higgins JM, Light DR. MetAP2 inhibition modifies hemoglobin S to delay polymerization and improves blood flow in sickle cell disease Blood Adv . 2021 2021 1388-1402 PubMed ID: 33661300 DOI: 10.1182/bloodadvances.2020003670

16065 Baik R, Wyman SK, Kabir S, Corn JE. Genome editing to model and reverse a prevalent mutation associated with myeloproliferative neoplasms PLoS One 2021 16(3):e0247858 PubMed ID: 33661998 DOI: 10.1371/journal.pone.0247858

16892 Uchida N, Drysdale CM, Nassehi T, Gamer J, Yapundich M, DiNicola J, Shibata Y, Hinds M, Gudmundsdottir B, Haro-Mora JJ, Demirci S, Tisdale JF. Cas9 protein delivery non-integrating lentiviral vectors for gene correction in sickle cell disease Mol Ther Methods Clin Dev 2021 21:121-132 PubMed ID: 33816645 DOI: 10.1016/j.omtm.2021.02.022

16895 Hua P, Badat M, Hanssen LLP, Hentges LD, Crump N, Downes DJ, Jeziorska DM, Oudelaar AM, Schwessinger R, Taylor S, Milne TA, Hughes JR, Higgs DR, Davies JOJ. Defining genome architecture at base-pair resolution Nature 2021 595(7865):125-129 PubMed ID: 34108683 DOI: 10.1038/s41586-021-03639-4

18386 Kevin R. Gillinder, Casie Leigh Reed, Shezlie Malelang, Helen Lorraine Mitchell, Emma Hoskin, Graham William Magor, Zane Kaplan, Andrew Charles Perkins Gene Editing of KLF1 to Cure Sickle Cell Disease Blood 2020 136:30-31 DOI: 10.1182/blood-2020-142339

18387 Divya S Vinjamur, Qiuming Yao, Mitchel A. Cole, Connor McGuckin, Chunyan Ren, Jing Zeng, Mir Hossain, Luca Pinello, Daniel E Bauer ZNF410 Represses Fetal Globin By Devoted Control of CHD4/NuRD Blood 2020 136:1-1 DOI: 10.1182/blood-2020-142134

18394 Georgia L. Gregory, Beeke Wienert, Marisa Schwab, Billie Rachael Lianoglou, Roger P. Hollis, Donald B. Kohn, Bruce R. Conklin, Tippi C. MacKenzie Investigating Zeta Globin Gene Expression to Develop a Potential Therapy for Alpha Thalassemia Major Blood 2020 136:3-4 DOI: 10.1182/blood-2020-142922

18429 Her NG, Babic I, Yenugonda VM, Kesari S, Nurmemmedov E. Cellular thermal shift analysis for interrogation of CRISPR-assisted proteomic changes Biotechniques 2020 68(4):180-184. PubMed ID: 32040335 DOI: 10.2144/btn-2019-0100

18432 Zhang X, Chen L, Zhu B, Wang L, Chen C, Hong M, Huang Y, Li H, Han H, Cai B, Yu W, Yin S, Yang L, Yang Z, Liu M, Zhang Y, Mao Z, Wu Y, Liu M, Li D. Increasing the efficiency and targeting range of cytidine base editors through fusion of a single-stranded DNA-binding protein domain Nat Cell Biol 2020 22(6):740-750 PubMed ID: 32393889 DOI: 10.1038/s41556-020-0518-8

18433 Pavani G, Laurent M, Fabiano A, Cantelli E, Sakkal A, Corre G, Lenting PJ, Concordet JP, Toueille M, Miccio A, Amendola M. Ex vivo editing of human hematopoietic stem cells for erythroid expression of therapeutic proteins Nat Commun 2020 11(1):3778 PubMed ID: 32728076 DOI: 10.1038/s41467-020-17552-3

18490 Boontanrart MY, Schröder MS, Stehli GM, Banović M, Wyman SK, Lew RJ, Bordi M, Gowen BG, DeWitt MA, Corn JE. ATF4 Regulates MYB to Increase γ-Globin in Response to Loss of β-Globin Cell Rep 2020 32(5):107993 PubMed ID: 32755585 DOI: 10.1016/j.celrep.2020.107993

18493 Karayel Ö, Xu P, Bludau I, Velan Bhoopalan S, Yao Y, Ana Rita FC, Santos A, Schulman BA, Alpi AF, Weiss MJ, Mann M. Integrative proteomics reveals principles of dynamic phosphosignaling networks in human erythropoiesis Mol Syst Biol 2020 16(12):e9813 PubMed ID: 33259127 DOI: 10.15252/msb.20209813

18505 Romano O, Petiti L, Felix T, Meneghini V, Portafax M, Antoniani C, Amendola M, Bicciato S, Peano C, Miccio A. GATA Factor-Mediated Gene Regulation in Human Erythropoiesis iScience 2020 23(4):101018 PubMed ID: 32283524 DOI: 10.1016/j.isci.2020.101018

6780 Makino T, Haruyama M, Katayama K, Terashima H, Tsunemi T, Miyazaki K, Terakawa M, Yamashiro K, Yoshioka R, Maeda H. Phenotypic-screening generates active novel fetal globin-inducers that downregulate Bcl11a in a monkey model. Biochem Pharmacol 2020 171:113717 PubMed ID: 31751536 DOI: 10.1016/j.bcp.2019.113717

8970 Leslie Weber, Giacomo Frati, Tristan Felix, Giulia Hardouin, Antonio Casini, Clara Wollenschlaeger, Vasco Meneghini, Cecile Masson, Anne De Cian, Anne Chalumeau, Fulvio Mavilio, Mario Amendola, Isabelle Andre-Schmutz, Anna Cereseto, Wassim El Nemer, Jean-Paul Concordet, Carine Giovannangeli, Marina Cavazzana, Annarita Miccio Editing a γ-Globin Repressor Binding Site Restores Fetal Hemoglobin Synthesis and Corrects the Sickle Cell Disease Phenotype Sci Adv 2020 6 (7), eaay9392 PubMed ID: 32917636 DOI: 10.1126/sciadv.aay9392

14505 Demirci S, Gudmundsdottir B, Li Q, Haro-Mora JJ, Nassehi T, Drysdale C, Yapundich M, Gamer J, Seifuddin F, Tisdale JF, Uchida N. βT87Q-Globin Gene Therapy Reduces Sickle Hemoglobin Production, Allowing for Ex Vivo Anti-sickling Activity in Human Erythroid Cells Mol Ther Methods Clin Dev 2020 17:912-921. PubMed ID: 32405513 DOI: 10.1016/j.omtm.2020.04.013

18405 Kumkhaek C, LaChance C, Aerbajinai W, Zhu J, Rodgers GP. Role of MFHAS1 in regulating hepcidin expression via the BMP/SMAD and MAPK/ERK1/2 signalling pathways Br J Haematol 2019 186(4):e108-e112 PubMed ID: 31049940 DOI: 10.1111/bjh.15939

18411 Lan X, Khandros E, Huang P, Peslak SA, Bhardwaj SK, Grevet JD, Abdulmalik O, Wang H, Keller CA, Giardine B, Baeza J, Duffner ER, El Demerdash O, Wu XS, Vakoc CR, Garcia BA, Hardison RC, Shi J, Blobel GA. The E3 ligase adaptor molecule SPOP regulates fetal hemoglobin levels in adult erythroid cells Blood ABlood Adv 2019 3(10):1586-1597. PubMed ID: 31126914 DOI: 10.1182/bloodadvances.2019032318

18427 Rivers A, Molokie R, Lavelle D. A new target for fetal hemoglobin reactivation Haematologica 2019 104(12):2325-2327 PubMed ID: 31787612 DOI: 10.3324/haematol.2019.230904

6745 Hyle J, Zhang Y, Wright S, Xu B, Shao Y, Easton J, Tian L, Feng R, Xu P, Li C. Acute depletion of CTCF directly affects MYC regulation through loss of enhancer-promoter looping. Nucleic Acids Res 2019 47(13):6699-6713 PubMed ID: 31127282 DOI: 10.1093/nar/gkz462

6766 Mudit Chaand, PhD , Chris Fiore, PhD , Brian T Johnston, MS , Diane H Moon, PhD , John P Carulli, PhD , Jeffrey R Shearstone, PhD Chromatin Accessibility Mapping of Primary Erythroid Cell Populations Leads to Identification and Validation of Nuclear Factor I X (NFIX) As a Novel Fetal Hemoglobin (HbF) Repressor Blood 2019 134 (Supplement_1): 812 DOI: 10.1182/blood-2019-124337

6767 Laura Breda, PhD , Valentina Ghiaccio, PhD , Silvia Pires Lourenco, MSc , Danuta Jadwiga Jarocha, PhD , Yasuhiro Ikawa, MD PhD , Naoto Tanaka, PhD , Virginia Guzikowski , Carla Casu, PhD , Kim Smith-Whitley, MD , Janet L. Kwiatkowski, MD MSCE , Stefano Rivella, PhD Preclinical Evaluation of ALS20, a New and Improved Lentiviral Vector for Beta-Globinopathies Blood 2019 134 (Supplement_1): 2242 DOI: 10.1182/blood-2019-124043

6769 Craig M Forester, MD PhD , Gun Woo-Byeon , Juan Oses-Prieto, PhD , Al Burlingame, PhD , Maria Barna, PhD , Davide M Ruggero, PhD Regulation of eIF4E Guides a Unique Translational Program to Steer Erythroid Maturation Blood 2019 134 (Supplement_1): 156 DOI: 10.1182/blood-2019-129608

6772 Kang J, Kang Y, Kim YW, You J, Kang J, Kim A. LRF acts as an activator and repressor of the human β-like globin gene transcription in a developmental stage dependent manner. Biochem Cell Biol 2019 97(4):380-386 PubMed ID: 30427207 DOI: 10.1139/bcb-2018-0303

6779 Sukanya Chumchuen, MSc, BSc , Tanapat Pornsukjantra, B.Sc. , Pinyaphat Khamphikham, PhD , Usanarat Anurathapan, MD , Orapan Sripichai, PhD Downregulation of Transcription Factor LRF/ZBTB7A Increases Fetal Hemoglobin Expression in β-Thalassemia/Hemoglobin E Erythroid Cells Blood 2019 134 (Supplement_1): 3549 DOI: 10.1182/blood-2019-126664

6783 Pamela Himadewi, BSc , Xiaotian Zhang, PhDBS , Haley Gore, MS , Xue Qing David Wang, PhD Chromosomal Loop Editing Reveals New Epigenomic Landscape in the β-Globin Locus with Implications for Hemoglobinopathy Therapies Blood 2019 134 (Supplement_1): 813 DOI: 10.1182/blood-2019-125020

6792 Zhou Z, Giles KE, Felsenfeld G. DNA·RNA triple helix formation can function as a cis-acting regulatory mechanism at the human β-globin locus. Proc Natl Acad Sci U S A 2019 116(13):6130-6139 PubMed ID: 30867287 DOI: 10.1073/pnas.1900107116

6794 Li C, Mishra AS, Gil S, Wang M, Georgakopoulou A, Papayannopoulou T, Hawkins RD, Lieber A. Targeted Integration and High-Level Transgene Expression in AAVS1 Transgenic Mice after In Vivo HSC Transduction with HDAd5/35++ Vectors. Mol Ther 2019 27(12):2195-2212 PubMed ID: 31494053 DOI: 10.1016/j.ymthe.2019.08.006

6797 Wang H, Liu Z, Li C, Gil S, Papayannopoulou T, Doering CB, Lieber A High-level protein production in erythroid cells derived from in vivo transduced hematopoietic stem cells. Blood Adv 2019 3(19):2883-2894 PubMed ID: 31585952 DOI: 10.1182/bloodadvances.2019000706

6801 Ludwig LS, Lareau CA, Bao EL, Nandakumar SK, Muus C, Ulirsch JC, Chowdhary K, Buenrostro JD, Mohandas N, An X, Aryee MJ, Regev A, Sankaran VG. Transcriptional States and Chromatin Accessibility Underlying Human Erythropoiesis. Cell Rep 2019 27(11):3228-3240.e7 PubMed ID: 31189107 DOI: 10.1016/j.celrep.2019.05.046

6810 Métais JY, Doerfler PA, Mayuranathan T, Bauer DE, Fowler SC, Hsieh MM, Katta V, Keriwala S, Lazzarotto CR, Luk K, Neel MD, Perry SS, Peters ST, Porter SN, Ryu BY, Sharma A, Shea D, Tisdale JF, Uchida N, Wolfe SA, Woodard KJ, Wu Y, Yao Y, Zeng J, Pruett-Miller S, Tsai SQ, Weiss MJ. Genome editing of HBG1 and HBG2 to induce fetal hemoglobin. Blood Adv 2019 3(21):3379-3392 PubMed ID: 31698466 DOI: 10.1182/bloodadvances.2019000820

6816 Papasavva P, Kleanthous M, Lederer CW. Rare Opportunities: CRISPR/Cas-Based Therapy Development for Rare Genetic Diseases. Mol Diagn Ther. 2019 23(2):201-222 PubMed ID: 30945166 DOI: 10.1007/s40291-019-00392-3

18193 Alicia Chang, So Hyun Park, Ciaran M Lee, Alireza Paikari, Gang Bao, Vivien A Sheehan Sickle Human Umbilical Cord Derived Erythroid Progenitor Cells (S-HUDEP2): An Ideal in-Vitro System for Screening Anti-Sickling Compounds for Sickle Cell Disease Blood 2018 132:3675-3675 DOI: 10.1182/blood-2018-99-117158

18395 Peng Xu, Daniel C. Scott, Xing Tang, Yu Yao, Yong-Dong Wang, Wenjian Bi, Lance E. Palmer, Ruopeng Feng, Arno F. Alpi, Chunliang Li, Yong Cheng, Brenda A. Schulman, Mitchell J. Weiss FBXO11 Activates Erythroid Gene Transcription By Degrading Heterochromatin-Associated Protein BAHD1 Blood 2018 132:529-529 DOI: 10.1182/blood-2018-99-117362

18397 Ruopeng Feng, Phillip A Doerfler, Yu Yao, Xing Tang, Yong-Dong Wang, Chunliang Li, David E. Root, Yong Cheng, Mitchell J. Weiss The DNA Methylation Maintenance Protein UHRF1 Regulates Fetal Globin Expression Independent of HBG Promoter DNA Methylation Blood 2018 132:410-410 DOI: 10.1182/blood-2018-99-115589

18407 Peter Balogh, Brian Capaldo, Sandeep Singh, Kamaleldin Elagib, Hui Li, Eli Zunder, Maria E. Figueroa, Wendy Pang, Adam Goldfarb Dysregulation of RUNX3 in Aged Human HSPCs May Contribute to Perturbations in Erythropoiesis and Balanced Lineage Output Blood 2018 132:2553-2553 DOI: 10.1182/blood-2018-99-115245

18496 Li C, Psatha N, Sova P, Gil S, Wang H, Kim J, Kulkarni C, Valensisi C, Hawkins RD, Stamatoyannopoulos G, Lieber A. Reactivation of γ-globin in adult β-YAC mice after ex vivo and in vivo hematopoietic stem cell genome editing Blood 2018 131(26):2915-2928 PubMed ID: 29789357 DOI: 10.1182/blood-2018-03-838540

18510 Xianjiang Lan, Eugene Khandros, Jeremy D. Grevet, Scott A. Peslak, Saurabh Bhardwaj, Cheryl A. Keller, Belinda Giardine, Ben A. Garcia, Ross C. Hardison, Junwei Shi, Gerd A. Blobel Domain-Focused CRISPR-Cas9 Screen Identifies the E3 Ubiquitin Ligase Substrate Adaptor Protein SPOP as a Novel Repressor of Fetal Hemoglobin Blood 2018 132:414-414 DOI: 10.1182/blood-2018-99-119518

4246 Schoonenberg VAC, Cole MA, Yao Q, Macias-Treviño C, Sher F, Schupp PG, Canver MC, Maeda T, Pinello L, Bauer DE. CRISPRO: identification of functional protein coding sequences based on genome editing dense mutagenesis. Genome Biol. 2018 PubMed ID: 30340514 DOI: 10.1186/s13059-018-1563-5

4518 Vinjamur DS, Bauer DE Growing and Genetically Manipulating Human Umbilical Cord Blood-Derived Erythroid Progenitor (HUDEP) Cell Lines. Erythropoiesis 2018 275-284 PubMed ID: 29076097 DOI: 10.1007/978-1-4939-7428-3_17

6782 Li C, Psatha N, Gil S, Wang H, Papayannopoulou T, Lieber A. HDAd5/35++ Adenovirus Vector Expressing Anti-CRISPR Peptides Decreases CRISPR/Cas9 Toxicity in Human Hematopoietic Stem Cells. Mol Ther Methods Clin Dev 2018 9:390-401 PubMed ID: 30038942 DOI: 10.1016/j.omtm.2018.04.008

6805 Nussbaum L, Telenius JM, Hill S, Hirschfeld PP, Suciu MC, WIGWAM Consortium, Downes DJ, Hughes JR. High-Throughput Genotyping of CRISPR/Cas Edited Cells in 96-Well Plates. Methods Protoc 2018 1(3):29 PubMed ID: 31164571 DOI: 10.3390/mps1030029

6806 Hsu JY, Fulco CP, Cole MA, Canver MC, Pellin D, Sher F, Farouni R, Clement K, Guo JA, Biasco L, Orkin SH, Engreitz JM, Lander ES, Joung JK, Bauer DE, Pinello L. CRISPR-SURF: discovering regulatory elements by deconvolution of CRISPR tiling screen data. Nat Methods 2018 15(12):992-993 PubMed ID: 30504875 DOI: 10.1038/s41592-018-0225-6

6812 Leung A, Zulick E, Skvir N, Vanuytsel K, Morrison TA, Naing ZH, Wang Z, Dai Y, Chui DHK, Steinberg MH, Sherr DH, Murphy GJ. Notch and Aryl Hydrocarbon Receptor Signaling Impact Definitive Hematopoiesis from Human Pluripotent Stem Cells. Stem Cells 2018 36(7):1004-1019 PubMed ID: 29569827 DOI: 10.1002/stem.2822

6822 Canver MC, Haeussler M, Bauer DE, Orkin SH, Sanjana NE, Shalem O, Yuan GC, Zhang F, Concordet JP, Pinello L Integrated design, execution, and analysis of arrayed and pooled CRISPR genome-editing experiments. Nat Protoc 2018 13(5):946-986 PubMed ID: 29651054 DOI: 10.1038/nprot.2018.005

18392 Xiaofei Yu, Alex Azzo, David C. Williams, Gordon Ginder MBD2-NuRD but Not MBD3-NuRD Mediates Strong Silencing of Fetal γ-Globin Gene Expression in Human Adult Erythroid Cells blood 2017 130:2220 DOI: 10.1182/blood.V130.Suppl_1.2220.2220

18404 Naoya Uchid, Anna Shvygin, Lydia Raines, Juan Jesus J. Haro Mora, Selami Demirci, Matthew M. Hsieh, John F Tisdale Selective Proliferation of Gene-Modified Human Erythroid Cells with Truncated Erythropoietin Receptors Blood 2017 130:3338 DOI: 10.1182/blood.V130.Suppl_1.3338.3338

6781 Maroofi N, Azarkeivan A, Banihashemi S, Mohammadparast S, Aghajanirefah A, Banan M. An enhancer haplotype may influence BCL11A expression levels and the response to hydroxyurea in β-thalassemia patients. Pharmacogenomics 2017 18(10):995-967 PubMed ID: 28639471 DOI: 10.2217/pgs-2017-0019

6800 Joung J, Konermann S, Gootenberg JS, Abudayyeh OO, Platt RJ, Brigham MD, Sanjana NE, Zhang F. Genome-scale CRISPR-Cas9 knockout and transcriptional activation screening. Nat Protoc 2017 12(4):828-863 PubMed ID: 28333914 DOI: 10.1038/nprot.2017.016

18412 Emily N Stern, Samuel Lessard, Patrick G. Schupp, Falak Sher, Guillaume Lettre, Daniel E. Bauer An Essential Erythroid-Specific Enhancer of ATP2B4 Associated with Red Blood Cell Traits and Malaria Susceptibility Blood 2016 128:1250-1250 DOI: 10.1182/blood.V128.22.1250.1250