Recapitulating muscle disease phenotypes with myotonic dystrophy 1 induced pluripotent stem cells: a tool for disease modeling and drug discovery

• Asıl Yazarlar: Ricardo Mondragon-Gonzalez (Yazar), Rita C. R. Perlingeiro (Yazar)
• Materyal Türü: Kitap
• Baskı/Yayın Bilgisi: The Company of Biologists, 2018-07-01T00:00:00Z.
• Konular: article
• Online Erişim: Connect to this object online.

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...