Both TALENs and CRISPR/Cas9 directly target the HBB IVS2–654 (C > T) mutation in β-thalassemia-derived iPSCs

β-Thalassemia is one of the most common genetic blood diseases and is caused by either point mutations or deletions in the β-globin (HBB) gene. The generation of patient-specific induced pluripotent stem cells (iPSCs) and subsequent correction of the disease-causing mutations may be a potential ther...

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Detalles Bibliográficos
Autores principales: Xu, Peng, Tong, Ying, Liu, Xiu-zhen, Wang, Ting-ting, Cheng, Li, Wang, Bo-yu, Lv, Xiang, Huang, Yue, Liu, De-pei
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2015
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4496796/
https://www.ncbi.nlm.nih.gov/pubmed/26156589
http://dx.doi.org/10.1038/srep12065