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Cell No. : Cell Name
RCB4557 : HUDEP-2  update : 2024/07/18
CommentHuman erythroid progenitor cell line derived from umbilical cord blood cells. Expressing adult type hemoglobin.
Comment from the depositor
Terms and conditions1) BIOLOGICAL RESOURCE is available only for academic research by not-for-profit organization. Regarding for-profit use and/or use by for-profit organization please contact us (cellbank.brc@riken.jp). 2) In publishing research results obtained by the use of the BIOLOGICAL RESOURCE, a citation of the original publication (PLoS One. 2013;8(3):e59890) designated by the DEPOSITOR is required.
Remarks
Order Form Order Form(C-0005.pdf)   MTA(C-0007.pdf)  
Regarding MTA between user institutions and RIKEN BRC, there are two kinds of MTA, not-for-profit academic purpose (C-XXXX) and for-profit research purpose (C-XXXXp) , depending on the sort of user institutions and the purposes of use. Please use an appropriate MTA(to see). In relation to commercial use and use for patent filing, first of all Please contact RIKEN BRC (cellbank.brc@riken.jp).
Basic information IPR RIKEN BioResource Research Center (Japan)
Depositor KURITA, Ryo
Originator KURITA, Ryo
Year of deposit 2013
Cloning (depositor) No
Animal _human < Mammals
Genus Homo
Species sapiens
Race Japanese
Tissue umbilical cord blood
Classification transformed
Recombinant recombinant
Exogene KUSABIRA-ORANGE, HPV16 E6/E7
Vector CSIV-TRE-HPV-E6/E7-UbC-KT lentiviral vector
Lifespan infinite
Morphology lymphocyte-like
Cellosaurus(Expasy) CVCL_VI06
deposit info
lot info
Medium Medium List
Culture type Suspension cells Suspension cells
Culture method 浮遊細胞の培養に関する一般的な注意(Japanese)
Culture medium StemSpan SFEM (Stemcell technologies), EPO(3U/ml), SCF(50ng/ml), DEX(10-6M), DOX(1ug/ml) StemSpan SFEM + 3U/ml EPO + 50ng/ml SCF + 10 -6M Dexamethasone + 1ug/ml Doxycycline
Antibiotics Free
Passage method dilution
Culture information Passage ratio 1 : 8-10 split
SC frequency Subculture : 2 times/week Subculture : 2 times/week
Temperature 37 ℃ 37 ℃
CO2 concentration 5 % 5 %
Freeze medium Medium + 10% DMSO CELLBANKER 1
Freezing method Slow freezing
Mycoplasma (-)
Animal PCR OK
Virus (HIV) Lot.1 : Undetected
Virus (HTLV-1) Lot.1 : (-)
STR(human) OK
deposit info
lot info
D5S818 D13S317 D7S820 D16S539 vWA TH01 Amelogenin TPOX CSF1PO
11,13 8,12 10,11 11,14 14,19 6,9 X,Y 8,11 10,13
Reference information Reference 163
User's Publication 120


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Reference
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
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  Curr Opin Hematol  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    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
6820  Cottle RN, Lee CM, Bao G.  Treating hemoglobinopathies using gene-correction approaches: promises and challenges.  Hum Genet  2016  135(9):993-1010  PubMed ID: 27314256   DOI: 10.1007/s00439-016-1696-0
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
18367  Takeshi Masuda, Xin Wang, Manami Maeda, Matthew C. Canver, Falak Sher, Alister P.W. Funnell, Chris Fisher, Maria Suciu, Gabriella E. Martyn, Laura J. Norton, Ruijia Zhu, Ryo Kurita, Yukio Nakamura, Jian Xu, Douglas R. Higgs, Merlin Crossley, Daniel E. Bauer, Stuart H. Orkin, Peter V. Kharchenko, Takahiro Maeda  The LRF/ZBTB7A Transcription Factor Is a BCL11A-Independent Repressor of Fetal Hemoglobin  Blood  2015  126:2-2    DOI: 10.1182/blood.V126.23.2.2
6756  Kim YH, Kim HO, Baek EJ, Kurita R, Cha HJ, Nakamura Y, Kim H.  Rh D blood group conversion using transcription activator-like effector nucleases.  Nat Commun  2015  6:7451  PubMed ID: 26078220   DOI: 10.1038/ncomms8451
6775  Canver MC, Smith EC, Sher F, Pinello L, Sanjana NE, Shalem O, Chen DD, Schupp PG, Vinjamur DS, Garcia SP, Luc S, Kurita R, Nakamura Y, Fujiwara Y, Maeda T, Yuan GC, Zhang F, Orkin SH, Bauer DE.  BCL11A enhancer dissection by Cas9-mediated in situ saturating mutagenesis.  Nature  2015  527(7577):192-7  PubMed ID: 26375006   DOI: 10.1038/nature15521
6742  Kurita R, Suda N, Sudo K, Miharada K, Hiroyama T, Miyoshi H, Tani K, Nakamura Y.  Establishment of immortalized human erythroid progenitor cell lines able to produce enucleated red blood cells.  PLoS One  2013  8(3):e59890  PubMed ID: 23533656   DOI: 10.1371/journal.pone.0059890

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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
18514  Daniels DE, Ferrer-Vicens I, Hawksworth J, Andrienko TN, Finnie EM, Bretherton NS, Ferguson DCJ, Oliveira ASF, Szeto JA, Wilson MC, Brewin JN, Frayne J.  Human cellular model systems of β-thalassemia enable in-depth analysis of disease phenotype  Nat Commun  2023  14(1):6260.  PubMed ID: 37803026   DOI: 10.1038/s41467-023-41961-9
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
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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
18448  Jiang F, Zuo L, Li D, Li J, Tang X, Chen G, Zhou J, Lu H, Liao C.  Molecular epidemiology and hematologic characterization of δβ-thalassemia and hereditary persistence of fetal hemoglobin in 125,661 families of greater Guangzhou area, the metropolis of southern China  BMC Med Genet  2020  21(1):43  PubMed ID: 32111191   DOI: 10.1186/s12881-020-0981-x
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
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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
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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
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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
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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
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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
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