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KLF1 Directly Activates Expression of the Novel Fetal Globin Repressor ZBTB7A/LRF in Erythroid Cells
Blood Adv
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Shadi Khalil, Maja Holy, Stephen Grado, Robert Fleming, Ryo Kurita, Yukio Nakamura, Adam Goldfarb
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Blood Adv
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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
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A genome-editing strategy to treat β-hemoglobinopathies that recapitulates a mutation associated with a benign genetic condition.
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Hum Mol Genet
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Hum Genet
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Nrf2 activation and fetal hemoglobin induction in sickle cell disease
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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
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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
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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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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
|
|
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
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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
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6810
Métais JY, Doerfler PA, Mayuranathan T, Bauer DE, Fowler SC, Hsieh MM, Katta V, Keriwala S, Lazzarotto CR, Luk K, Neel MD, Perry SS, Peters ST, Porter SN, Ryu BY, Sharma A, Shea D, Tisdale JF, Uchida N, Wolfe SA, Woodard KJ, Wu Y, Yao Y, Zeng J, Pruett-Miller S, Tsai SQ, Weiss MJ.
Genome editing of HBG1 and HBG2 to induce fetal hemoglobin.
Blood Adv
2019
3(21):3379-3392
PubMed ID: 31698466
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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
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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
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4518
Vinjamur DS, Bauer DE
Growing and Genetically Manipulating Human Umbilical Cord Blood-Derived Erythroid Progenitor (HUDEP) Cell Lines.
Erythropoiesis
2018
275-284
PubMed ID: 29076097
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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
|
|
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
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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
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|
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
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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
|
|
6814
Vivien A. C. Schoonenberg, Mitchel A. Cole, Qiuming Yao, Claudio Macias-Treviño, Falak Sher, Patrick G. Schupp, Matthew C. Canver, Takahiro Maeda, Luca Pinello, Daniel E. Bauer
CRISPRO Identifies Functional Protein Coding Sequences Based on Genome Editing Dense Mutagenesis
bioRxiv
2018
|
|
6822
Canver MC, Haeussler M, Bauer DE, Orkin SH, Sanjana NE, Shalem O, Yuan GC, Zhang F, Concordet JP, Pinello L
Integrated design, execution, and analysis of arrayed and pooled CRISPR genome-editing experiments.
Nat Protoc
2018
13(5):946-986
PubMed ID: 29651054
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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
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|
6800
Joung J, Konermann S, Gootenberg JS, Abudayyeh OO, Platt RJ, Brigham MD, Sanjana NE, Zhang F.
Genome-scale CRISPR-Cas9 knockout and transcriptional activation screening.
Nat Protoc
2017
12(4):828-863
PubMed ID: 28333914
|
|
6802
Wright JB, Sanjana NE.
CRISPR Screens to Discover Functional Noncoding Elements.
Trends Genet
2016
32(9):526-529
PubMed ID: 27423542
|
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