Genome Modification Leads to Phenotype Reversal in Human Myotonic Dystrophy type 1 iPS-cell Derived Neural Stem Cells

OBJECTIVE: Myotonic dystrophy type 1 (DM1) is caused by expanded CTG repeats in the 3'-untranslated region (3’ UTR) of the DMPK gene. Correcting the mutation in DM1 stem cells would be an important step towards autologous stem cell therapy. The objective of this study is to demonstrate in vitro...

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Detalhes bibliográficos
Publicado no:Stem Cells
Main Authors: Xia, Guangbin, Gao, Yuanzheng, Jin, Shouguang, Subramony, SH., Terada, Naohiro, Ranum, Laura P.W., Swanson, Maurice S., Ashizawa, Tetsuo
Formato: Artigo
Idioma:Inglês
Publicado em: 2015
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4441571/
https://ncbi.nlm.nih.gov/pubmed/25702800
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/stem.1970
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