Disease modeling and lentiviral gene transfer in patient-specific induced pluripotent stem cells from late-onset Pompe disease patient

Pompe disease is an autosomal recessive inherited metabolic disease caused by deficiency of acid α-glucosidase (GAA). Glycogen accumulation is seen in the affected organ such as skeletal muscle, heart, and liver. Hypertrophic cardiomyopathy is frequently seen in the infantile onset Pompe disease. On...

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Detalhes bibliográficos
Publicado no:Mol Ther Methods Clin Dev
Main Authors: Sato, Yohei, Kobayashi, Hiroshi, Higuchi, Takashi, Shimada, Yohta, Era, Takumi, Kimura, Shigemi, Eto, Yoshikatsu, Ida, Hiroyuki, Ohashi, Toya
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2015
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4495721/
https://ncbi.nlm.nih.gov/pubmed/26199952
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mtm.2015.23
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