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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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Detalhes bibliográficos
Publicado no:Dis Model Mech
Main Authors: Mondragon-Gonzalez, Ricardo, Perlingeiro, Rita C. R.
Formato: Artigo
Idioma:Inglês
Publicado em: The Company of Biologists Ltd 2018
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6078411/
https://ncbi.nlm.nih.gov/pubmed/29898953
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1242/dmm.034728
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