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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...

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Detalles Bibliográficos
Autores principales: Mondragon-Gonzalez, Ricardo, Perlingeiro, Rita C. R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: The Company of Biologists Ltd 2018
Materias:
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

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