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

In the present report, we carried out clinical-scale editing in adult mobilized CD34+ hematopoietic stem and progenitor cells (HSPCs) using zinc-finger nuclease-mediated disruption of BCL11a to upregulate the expression of γ-globin (fetal hemoglobin). In these cells, disruption of the erythroid-spec...

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Detalles Bibliográficos
Autores principales: Psatha, Nikoletta, Reik, Andreas, Phelps, Susan, Zhou, Yuanyue, Dalas, Demetri, Yannaki, Evangelia, Levasseur, Dana N., Urnov, Fyodor D., Holmes, Michael C., Papayannopoulou, Thalia
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2018
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6120587/
https://www.ncbi.nlm.nih.gov/pubmed/30182035
http://dx.doi.org/10.1016/j.omtm.2018.08.003

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6120587/
https://www.ncbi.nlm.nih.gov/pubmed/30182035
http://dx.doi.org/10.1016/j.omtm.2018.08.003

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