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

[31] Simvastatin and t-butylhydroquinone suppress KLF1 and BCL11A gene expression and additively increase fetal hemoglobin in primary human erythroid cells
Macari, Elizabeth R.
Schaeffer, Emily K.
West, Rachel J.
Lowrey, Christopher H.
BLOOD, 2013, 121 (05) : 830 - 839
[32] SCF-mediated γ-globin gene expression in adult human erythroid cells is associated with KLF1, BCL11A and SOX6 down-regulation
Lulli, Valentina
Romania, Paolo
Morsilli, Ornella
Ilari, Ramona
Gabbianelli, Marco
Testa, Ugo
Marziali, Giovanna
BLOOD CELLS MOLECULES AND DISEASES, 2015, 54 (01) : 1 - 3
[33] 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
[34] Multiplex CRISPR/Cas9 Genome Editing Targeting the BCL11A/HBG Axis Maximizes Fetal Hemoglobin Reinduction but Generates Chromosomal Translocations Which Persist In Vivo
Samuelson, Clare
Humbert, Olivier
Radtke, Stefan
Zhu, Haiying
Llewellyn, Mallory J.
Fields, Emily
Cook, Savannah
Huang, Meei-Li W.
Negre, Olivier
Jerome, Keith
Kiem, Hans-Peter
BLOOD, 2020, 136
[35] Clonal Analysis of Human Bone Marrow CD34+Cells Edited By BCL11A-Targeting Zinc Finger Nucleases Reveals Clinically Relevant Levels of Fetal Globin Expression in Edited Erythroid Progeny
Chang, Kai-Hsin
Sullivan, Timothy
Liu, Mei
Yang, Xiao
Sun, Chao
Vieira, Benjamin
Zhang, Ming
Long, Vu P.
Chen, Kai
Smith, Sarah
Tan, Siyuan
Reik, Andreas
Urnov, Fyodor U.
Rebar, Edward J.
Danos, Olivier
Jiang, Haiyan
BLOOD, 2015, 126 (23)
[36] Induction of Fetal Hemoglobin and Reduction of Clinical Manifestations in Patients with Severe Sickle Cell Disease Treated with Shmir-Based Lentiviral Gene Therapy for Post-Transcriptional Gene Editing of BCL11A: Updated Results from Pilot and Feasibility Trial
Esrick, Erica B.
Federico, Amy
Abriss, Daniela
Armant, Myriam
Boardman, Kari
Brendel, Christian
Ciuculescu, Marioara-Felicia
Daley, Heather
Dansereau, Colleen
Fernandes, Augustine
Heeney, Matthew M.
Justus, David G.
Kao, Pei-Chi
Kim, Annette S.
Kohn, Donald B.
Lehmann, Leslie E.
Liu, Donghui
London, Wendy B.
Manis, John P.
Moore, Theodore B.
Morris, Emily
Pellin, Danilo
Proeung, Ellen
Richard, Shanna
Roach, Gavin D.
Shaw, Kit L.
Terrazas, Dayna
White, Shanna L.
Williams, David A.
BLOOD, 2022, 140 : 10665 - 10667

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