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 th...

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Ngā kaituhi matua: Ricardo Mondragon-Gonzalez (Author), Rita C. R. Perlingeiro (Author)
Hōputu: Pukapuka
I whakaputaina: The Company of Biologists, 2018-07-01T00:00:00Z.
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