Comparative Studies Reveal Robust HbF Induction By Editing of HBG1/2 Promoters or BCL11A Erythroid-Enhancer in Human CD34+Cells but That BCL11A Erythroid-Enhancer Editing Is Associated with Selective Reduction in Erythroid Lineage Reconstitution in a Xenotransplantation Model

被引：6

作者：

Chang, Kai-Hsin ^{[1
]}

Sanchez, Minerva ^{[1
]}

Heath, Jack ^{[1
]}

dedreuzy, Edouard ^{[1
]}

Haskett, Scott ^{[1
]}

Vogelaar, Abigail ^{[1
]}

Gogi, Kiran ^{[1
]}

Da Silva, Jen ^{[1
]}

Wang, Tongyao ^{[1
]}

Sadowski, Andrew ^{[1
]}

Gotta, Gregory ^{[1
]}

Siwak, Jamaica ^{[1
]}

Viswanathan, Ramya ^{[1
]}

Loveluck, Katherine ^{[1
]}

Chao, Hoson ^{[1
]}

Tillotson, Eric ^{[1
]}

Chalishazar, Aditi ^{[1
]}

Dass, Abhishek ^{[1
]}

Ta, Frederick ^{[1
]}

Brennan, Emily ^{[1
]}

Tabbaa, Diana ^{[1
]}

Marco, Eugenio ^{[1
]}

Zuris, John A. ^{[1
]}

Reyon, Deepak ^{[1
]}

Isik, Meltem ^{[1
]}

Friedland, Ari ^{[1
]}

Ta, Terence ^{[1
]}

Harbinski, Fred ^{[1
]}

Giannoukos, Georgia ^{[1
]}

Teixeira, Sandra ^{[1
]}

Wilson, Christopher J. ^{[1
]}

Albright, Charlie ^{[1
]}

Jiang, Haiyan ^{[1
]}

机构：

[1] Editas Med Inc, Cambridge, MA USA

来源：

BLOOD | 2018年 / 132卷

关键词：

D O I：

10.1182/blood-2018-99-114780

中图分类号：

R5 [内科学];

学科分类号：

1002 ; 100201 ;

摘要：

409

引用

页数：3

共 5 条

[1] Fine-Mapping and Genome Editing Reveal An Essential Erythroid Enhancer At The HbF-Associated BCL11A Locus
Bauer, Daniel E.
Kamran, Sophia C.
Lessard, Samuel
Xu, Jian
Fujiwara, Yuko
Lin, Carrie
Shao, Zhen
Canver, Matthew C.
Smith, Elenoe C.
Pinello, Luca
Sabo, Peter J.
Vierstra, Jeff
Voit, Richard A.
Yuan, Guo-Cheng
Porteus, Matthew H.
Stamatoyannopoulos, John
Lettre, Guillaume
Orkin, Stuart H.
BLOOD, 2013, 122 (21)
[2] Genome editing of the BCL11A erythroid-specific enhancer in bone marrow-derived CD341 cells for the treatment of hemoglobinopathies
Chang, K. H.
Tan, S.
Smith, S.
Chen, K.
Sullivan, T.
Reik, A.
Urnov, F.
Rebar, E.
Gregory, P.
Danos, O.
Jiang, H.
HUMAN GENE THERAPY, 2015, 26 (10) : A7 - A7
[3] Durable and Robust Fetal Globin Induction without Anemia in Rhesus Monkeys Following Autologous Hematopoietic Stem Cell Transplant with BCL11A Erythroid Enhancer Editing
Demirci, Selami
Zeng, Jing
Wu, Yuxuan
Uchida, Naoya
Gamer, Jackson
Yapundich, Morgan
Drysdale, Claire
Bonifacino, Aylin C.
Krouse, Allen E.
Linde, Nathaniel S.
Donahue, Robert E.
Shen, Anne H.
Haro-Mora, Juan J.
Leonard, Alexis
Nassehi, Tina
Luk, Kevin
Wolfe, Scot A.
Lazzarotto, Cicera R.
Tsai, Shengdar Q.
Weiss, Mitchell J.
Bauer, Daniel E.
Tisdale, John F.
BLOOD, 2019, 134
[4] Ex Vivo Gene-Edited Cell Therapy for Sickle Cell Disease: Disruption of the BCL11A Erythroid Enhancer with Zinc Finger Nucleases Increases Fetal Hemoglobin in Plerixafor Mobilized Human CD34+Cells
Moran, Kevin
Ling, Hui
Lessard, Samuel
Vieira, Benjamin
Vu Hong
Holmes, Michael C.
Reik, Andreas
Dang, Denny
Gray, David
Levasseur, Dana
Rimmele, Pauline
BLOOD, 2018, 132
[5] Zinc Finger Nuclease-Mediated Disruption of the BCL11A Erythroid Enhancer in Human Hematopoietic Stem and Progenitor Cells Results in Enriched Bialleleic Editing with Highly Replicable and Precise On-Target Small Indels and Allele-Additive Increases in Fetal Hemoglobin
Ling, Hui
Lessard, Samuel
Vieira, Benjamin
Moran, Kevin
Rimmele, Pauline
Lin, Yi-Dong
Hong, Vu
Reik, Andreas
Dang, Denny
Huttner, Kenneth
Harris, Tim
Hicks, Alexandra
MOLECULAR THERAPY, 2019, 27 (04) : 449 - 449

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