Long-Term Engraftment and Fetal Globin Induction upon BCL11A Gene Editing in Bone-Marrow-Derived CD34(+) Hematopoietic Stem and Progenitor Cells

To develop an effective and sustainable cell therapy for sickle cell disease (SCD), we investigated the feasibility of targeted disruption of the BCL11A gene, either within exon 2 or at the GATAA motif in the intronic erythroid-specific enhancer, using zinc finger nucleases in human bone marrow (BM)...

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Detalles Bibliográficos
Autores principales: Chang, Kai-Hsin, Smith, Sarah E., Sullivan, Timothy, Chen, Kai, Zhou, Qianhe, West, Jason A., Liu, Mei, Liu, Yingchun, Vieira, Benjamin F., 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
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2017
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5363298/
https://www.ncbi.nlm.nih.gov/pubmed/28344999
http://dx.doi.org/10.1016/j.omtm.2016.12.009

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5363298/
https://www.ncbi.nlm.nih.gov/pubmed/28344999
http://dx.doi.org/10.1016/j.omtm.2016.12.009

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