Efficient Gene Editing at Major CFTR Mutation Loci

Cystic fibrosis (CF) is a lethal autosomal recessive disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Nuclease-mediated precise gene editing (PGE) represents a promising therapy for CF, for which an efficient strategy that is free of viral vector, d...

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Detalles Bibliográficos
Autores principales: Ruan, Jinxue, Hirai, Hiroyuki, Yang, Dongshan, Ma, Linyuan, Hou, Xia, Jiang, Hong, Wei, Hongguang, Rajagopalan, Carthic, Mou, Hongmei, Wang, Guoshun, Zhang, Jifeng, Li, Kui, Chen, Yuqing E., Sun, Fei, Xu, Jie
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6409404/
https://www.ncbi.nlm.nih.gov/pubmed/30852378
http://dx.doi.org/10.1016/j.omtn.2019.02.006

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