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Recapitulating muscle disease phenotypes with myotonic dystrophy 1 induced pluripotent stem cells: a tool for disease modeling and drug discovery
Myotonic dystrophy 1 (DM1) is a multisystem disorder primarily affecting the central nervous system, heart and skeletal muscle. It is caused by an expansion of the CTG trinucleotide repeats in the 3′ untranslated region of the DMPK gene. Although patient myoblasts have been used for studying the dis...
| Autores principales: | , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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The Company of Biologists Ltd
2018
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| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6078411/ https://www.ncbi.nlm.nih.gov/pubmed/29898953 http://dx.doi.org/10.1242/dmm.034728 |