Precise Correction of the Dystrophin Gene in Duchenne Muscular Dystrophy Patient Induced Pluripotent Stem Cells by TALEN and CRISPR-Cas9

Duchenne muscular dystrophy (DMD) is a severe muscle-degenerative disease caused by a mutation in the dystrophin gene. Genetic correction of patient-derived induced pluripotent stem cells (iPSCs) by TALENs or CRISPR-Cas9 holds promise for DMD gene therapy; however, the safety of such nuclease treatm...

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Gepubliceerd in:Stem Cell Reports
Hoofdauteurs: Li, Hongmei Lisa, Fujimoto, Naoko, Sasakawa, Noriko, Shirai, Saya, Ohkame, Tokiko, Sakuma, Tetsushi, Tanaka, Michihiro, Amano, Naoki, Watanabe, Akira, Sakurai, Hidetoshi, Yamamoto, Takashi, Yamanaka, Shinya, Hotta, Akitsu
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Elsevier 2014
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Online toegang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4297888/
https://ncbi.nlm.nih.gov/pubmed/25434822
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.stemcr.2014.10.013
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