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Length-dependent CTG·CAG triplet-repeat expansion in myotonic dystrophy patient-derived induced pluripotent stem cells

Myotonic dystrophy type 1 (DM1) is an inherited dominant muscular dystrophy caused by expanded CTG·CAG triplet repeats in the 3′ untranslated region of the DMPK1 gene, which produces a toxic gain-of-function CUG RNA. It has been shown that the severity of disease symptoms, age of onset and progressi...

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Autors principals: Du, Jintang, Campau, Erica, Soragni, Elisabetta, Jespersen, Christine, Gottesfeld, Joel M.
Format: Artigo
Idioma:Inglês
Publicat: Oxford University Press 2013
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Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC3842182/
https://ncbi.nlm.nih.gov/pubmed/23933738
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddt386
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