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Cell No. : Cell Name
RCB4557 : HUDEP-2  update : 2020/08/24
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 Regarding MTA between user institutions and RIKEN BRC, there are two kinds of MTA, Category I and II, depending on the sort of user institutions and the purposes of use.Please use an appropriate MTA(to see).In case of Restriction "a" or "f", please contact RIKEN BRC(cellbank.brc@riken.jp) regarding any kind of for-profit use.
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
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 : 5-10 split
SC frequency Subculture : 2 times/week Subculture : 2 times/week
Temperature 37 ℃ 37 ℃
CO2 concentration 5 % 5 %
Freeze medium Medium + 10% DMSO CELLBANKER 2
Freezing method Slow freezing
Mycoplasma (-)
STR(human) OK
Animal PCR OK
Virus (HIV) Undetected
Virus (HTLV-1) (-)
Reference information Reference 68
User's Publication 26


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Reference
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
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
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
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
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    PubMed ID: 32107331
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
9632  Selami Demirci et al  βT87Q-globin gene therapy reduces sickle hemoglobin production, allowing for ex vivo anti-sickling activity in human erythroid cells  Molecular Therapy: Methods & Clinical Development  2020   
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
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
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 
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 
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 
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 
6776  Orkin SH, Bauer DE.  Emerging Genetic Therapy for Sickle Cell Disease  Annu Rev Med  2019  70:257-271  PubMed ID: 30355263
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
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
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
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   
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   
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
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
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
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 
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
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
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
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
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
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
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
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
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
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
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
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
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   
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
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
6819  Paikari A, Sheehan VA.  Fetal haemoglobin induction in sickle cell disease.  Br J Haematol  2018  180(2):189-200  PubMed ID: 29143315
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 
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 
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 
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
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
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
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
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
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
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
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 
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 
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
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
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 
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
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
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
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
6804  Prakash V, Moore M, Yáñez-Muñoz RJ.  Current Progress in Therapeutic Gene Editing for Monogenic Diseases.  Mol Ther  2016  24(3):465-74  PubMed ID: 26765770
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
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 
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
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
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
9631  Richard A. Morgan et al  Creating New β-globin-Expressing Lentiviral Vectors by High-Resolution Mapping of Locus Control Region Enhancer Sequences  Molecular Therapy: Methods & Clinical Development  2010   
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

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User's Publication
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
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
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 
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 
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 
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
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 
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 
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
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