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Cell No. : Cell Name
RCB4557 : HUDEP-2  update : 2022/12/02
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 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
Medium and additives 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^(-6)M 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
Reference information Reference 77
User's Publication 42


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Reference
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
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
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
8969  Cristina Fugazza, Gloria Barbarani, Sudharshan Elangovan, Maria Giuseppina Marini, Serena Giolitto, Isaura Font-Monclus, Maria Franca Marongiu, Laura Manunza, John Strouboulis, Claudio Cantù, Fabio Gasparri, Silvia M L Barabino, Yukio Nakamura, Sergio Ottolenghi, Paolo Moi, Antonella Ellena Ronchi  The Coup-TFII Orphan Nuclear Receptor Is an Activator of the γ-Globin Gene  Haematologica  2020  106(2):474-482  PubMed ID: 32107331   DOI: 10.3324/haematol.2019.241224
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: 32095533   DOI: 10.1126/sciadv.aay9392
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
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   
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
6809  Ron Baik, Stacia K. Wyman, Shaheen Kabir, Jacob E. Corn1   Genome editing to model and reverse a prevalent mutation associated with myeloproliferative   bioRxiv  2019      DOI: 10.1101/855452
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
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
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
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
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
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
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   
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
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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
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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
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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
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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  02018  131 (17), 1960-1973  PubMed ID: 29519807   DOI: 10.1182/blood-2017-10-811505

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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
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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
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
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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
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
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
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
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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
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
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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
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
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
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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
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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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