Genome Therapy of Myotonic Dystrophy Type 1 iPS Cells for Development of Autologous Stem Cell Therapy

Myotonic dystrophy type 1 (DM1) is caused by expanded Cytosine-Thymine-Guanine (CTG) repeats in the 3′-untranslated region (3′ UTR) of the Dystrophia myotonica protein kinase (DMPK) gene, for which there is no effective therapy. The objective of this study is to develop genome therapy in human DM1 i...

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Publicat a:Mol Ther
Autors principals: Gao, Yuanzheng, Guo, Xiuming, Santostefano, Katherine, Wang, Yanlin, Reid, Tammy, Zeng, Desmond, Terada, Naohiro, Ashizawa, Tetsuo, Xia, Guangbin
Format: Artigo
Idioma:Inglês
Publicat: Nature Publishing Group 2016
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Original Article
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC5023370/
https://ncbi.nlm.nih.gov/pubmed/27203440
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2016.97
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