From GWAS To the Clinic: Genome-Editing the Human BCL11A Erythroid Enhancer for Fetal Globin Elevation in the Hemoglobinopathies

被引：0

作者：

Reik, Andreas ^{[1
]}

Chang, Kai-Hsin ^{[2
]}

Vierstra, Jeff ^{[2
]}

Psatha, Nikoletta ^{[2
]}

Stehling-Sun, Sandra ^{[2
]}

Zhou, Yuanyue ^{[1
]}

O'Neil, Colleen M. ^{[1
]}

Mich, Andrea ^{[1
]}

Miller, Jeffrey C. ^{[1
]}

Lee, Gary ^{[1
]}

Tan, Siyuan ^{[3
]}

Jiang, Haiyan ^{[3
]}

Stamatoyannopoulos, George ^{[2
]}

Papayannopoulou, Thalia ^{[2
]}

Rebar, Edward J. ^{[1
]}

Gregory, Philip D. ^{[1
]}

Stamatoyannopoulos, John A. ^{[2
]}

Urnov, Fyodor D. ^{[1
]}

机构：

[1] Sangamo BioSci Inc, Richmond, CA USA

[2] Univ Washington, Seattle, WA 98195 USA

[3] Biogen Idec Inc, Cambridge, MA USA

来源：

MOLECULAR THERAPY | 2015年 / 23卷

关键词：

D O I：

10.1016/S1525-0016(16)33658-9

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

引用

页码：S23 / S24

页数：2

共 36 条

[21] Preliminary Steps Toward Fetal Gene Therapy: Disruption of Bcl11a Erythroid-Specific Enhancer Region with CRISPR/Cas9
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Seferovic, Maxim
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Shanahan, Matthew
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Shamshirsaz, Alireza
Aagaard, Kjersti
REPRODUCTIVE SCIENCES, 2022, 29 (SUPPL 1) : 93 - 93
[22] CRISPR/Cas9-based multiplex genome editing of BCL11A and HBG efficiently induces fetal hemoglobin expression
Han, Yuanyuan
Tan, Xiaoyu
Jin, Tingting
Zhao, Siqi
Hu, Li
Zhang, Wei
Kurita, Ryo
Nakamura, Yukio
Liu, Juan
Li, Di
Zhang, Zhaojun
Fang, Xiangdong
Huang, Shengwen
EUROPEAN JOURNAL OF PHARMACOLOGY, 2022, 918
[23] CRISPR-Cas9 Genome Editing of γ-Globin Promoters in Human Hematopoietic Stem Cells to Induce Erythrocyte Fetal Hemoglobin for Treatment of β-Hemoglobinopathies
Metais, Jean-Yves
Doerfler, Phillip A.
Mayuranathan, Thiyagaraj
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Fowler, Stephanie
Hsieh, Matthew
Katta, Varun
Keriwala, Sagar
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Luk, Kevin
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Uchida, Naoya
Wu, Yuxuan
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Weiss, Mitchell J.
BLOOD, 2019, 134
[24] Induction of adult levels of β-globin in human erythroid cells that intrinsically express embryonic or fetal globin by transduction with KLF1 and BCL11A-XL
Trakarnsanga, Kongtana
Wilson, Marieangela C.
Lau, Winnie
Singleton, Belinda K.
Parsons, Steve F.
Sakuntanaga, Punthita
Kurita, Ryo
Nakamura, Yukio
Anstee, David J.
Frayne, Jan
HAEMATOLOGICA, 2014, 99 (11) : 1677 - 1685
[25] Combined+58 and+55 BCL11A enhancer Editing Yields Exceptional Efficiency, Specificity and HbF Induction in Human and NHP Preclinical Models
Zeng, Jing
Demirci, Selami
Nguyen, My Anh
Lin, Linda Yingqi
Maitland, Stacy A.
Mintzer, Esther
Wu, Yuxuan
Pellin, Danilo
Tangprasittipap, Amornrat
Vong, Chokdee
Porter, Shaina N.
Luk, Kevin
Liu, Pengpeng
Katta, Varun
Ciuculescu, Marioara-Felicia
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Hsu, Jonathan
Uchida, Naoya
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Hongeng, Suradej
Armant, Myriam
Wolfe, Scot A.
Tisdale, John F.
Bauer, Daniel E.
BLOOD, 2021, 138 : 1852 - +
[26] Combined+58 and+55 BCL11A Enhancer Editing Yields Exceptional Efficiency, Specificity and HbF Induction in Human Hematopoietic Stem Cells
Zeng, Jing
Nguyen, My Anh
Ciuculescu, Marioara-Felicia
Mintzer, Esther
Liu, Pengpeng
Lin, Linda
Rosanwo, Tolulope
Verma, Archana
Neri, Nola
Maitland, Stacy
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Brendel, Christian
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Manis, John
Armant, Myriam
Wolfe, Scot
Bauer, Daniel
MOLECULAR THERAPY, 2023, 31 (04) : 611 - 611
[27] Association between Variants at BCL11A Erythroid-Specific Enhancer and Fetal Hemoglobin Levels among Sickle Cell Disease Patients in Cameroon: Implications for Future Therapeutic Interventions
Pule, Gift Dineo
Bitoungui, Valentina Josiane Ngo
Chemegni, Bernard Chetcha
Kengne, Andre Pascal
Antonarakis, Stylianos
Wonkam, Ambroise
OMICS-A JOURNAL OF INTEGRATIVE BIOLOGY, 2015, 19 (10) : 627 - 631
[28] 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
[29] Transcription factors (TFs) negatively regulating γ-globin gene transcription: BCL11A is down-regulated during mithramycin induction of erythroid cells from β-thalassemia patients
Finotti, Alessia
Bianchi, Nicoletta
Zuccato, Cristina
Breveglieri, Giulia
Lampronti, Ilaria
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Gamberini, Maria Rita
Borgatti, Monica
Gambari, Roberto
INTERNATIONAL JOURNAL OF MOLECULAR MEDICINE, 2012, 30 : S60 - S60
[30] Long-Term Engraftment and Fetal Globin Induction upon BCL11A Gene Editing inBone-Marrow-Derived CD34+ Hematopoietic Stem and Progenitor Cells
Chang, Kai-Hsin
Smith, Sarah E.
Sullivan, Timothy
Chen, Kai
Zhou, Qianhe
West, Jason A.
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Sun, Chao
Hong, Vu P.
Zhang, Mingxuan
Yang, Xiao
Reik, Andreas
Urnov, Fyodor D.
Rebar, Edward J.
Holmes, Michael C.
Danos, Olivier
Jiang, Haiyan
Tan, Siyuan
MOLECULAR THERAPY-METHODS & CLINICAL DEVELOPMENT, 2017, 4 : 137 - 148

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