Disruption of the BCL11A Erythroid Enhancer Reactivates Fetal Hemoglobin in Erythroid Cells of Patients with β-Thalassemia Major-Reference-Cited by-同舟云学术

Disruption of the BCL11A Erythroid Enhancer Reactivates Fetal Hemoglobin in Erythroid Cells of Patients with β-Thalassemia Major

Published:2018-09 Issue: Volume:10 Page:313-326
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Psatha Nikoletta,Reik Andreas,Phelps Susan,Zhou Yuanyue,Dalas Demetri,Yannaki Evangelia,Levasseur Dana N.,Urnov Fyodor D.,Holmes Michael C.,Papayannopoulou Thalia

Funder

Cooley's Anemia Foundation

National Institutes of Health

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference50 articles.

1. Thalassaemia;Higgs;Lancet,2012

2. Gene Therapy of the β-Hemoglobinopathies by Lentiviral Transfer of the β(A(T87Q))-Globin Gene;Negre;Hum. Gene Ther.,2016

3. Update of Results from the Northstar Study (HGB-204): A Phase 1/2 Study of Gene Therapy for Beta-Thalassemia Major Via Transplantation of Autologous Hematopoietic Stem Cells Transduced Ex-Vivo with a Lentiviral Beta AT87Q-Globin Vector (LentiGlobin BB305);Walters;Blood,2015

4. Efficient Ex Vivo Engineering and Expansion of Highly Purified Human Hematopoietic Stem and Progenitor Cell Populations for Gene Therapy;Zonari;Stem Cell Reports,2017

5. Forced chromatin looping raises fetal hemoglobin in adult sickle cells to higher levels than pharmacologic inducers;Breda;Blood,2016

Cited by 94 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Delivery of nucleic acid based genome editing platforms via lipid nanoparticles: Clinical applications;Advanced Drug Delivery Reviews;2024-08

2. Precise correction of a spectrum of β-thalassemia mutations in coding and non-coding regions by base editors;Molecular Therapy - Nucleic Acids;2024-06

3. let-7 miRNAs repress HIC2 to regulate BCL11A transcription and hemoglobin switching;Blood;2024-05-09

4. Detection of CRISPR/Cas9-Mediated Fetal Hemoglobin Reactivation in Erythroblasts Derived from Cord Blood-Hematopoietic Stem Cells;Molecular Biotechnology;2024-04-22

5. Bone marrow Tfr2 deletion improves the therapeutic efficacy of the activin‐receptor ligand trap RAP‐536 in β‐thalassemic mice;American Journal of Hematology;2024-04-17