One-Step Biallelic and Scarless Correction of a β-Thalassemia Mutation in Patient-Specific iPSCs without Drug Selection

Monogenic disorders (MGDs), which are caused by single gene mutations, have a serious effect on human health. Among these, β-thalassemia (β-thal) represents one of the most common hereditary hematological diseases caused by mutations in the human hemoglobin β (HBB) gene. The technologies of induced...

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Detalles Bibliográficos
Autores principales: Liu, Yali, Yang, Yi, Kang, Xiangjin, Lin, Bin, Yu, Qian, Song, Bing, Gao, Ge, Chen, Yaoyong, Sun, Xiaofang, Li, Xiaoping, Bu, Lei, Fan, Yong
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/PMC5363452/
https://www.ncbi.nlm.nih.gov/pubmed/28325300
http://dx.doi.org/10.1016/j.omtn.2016.11.010

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