Pluripotent Stem Cells in Disease Modeling and Drug Discovery for Myotonic Dystrophy Type 1

Pluripotent Stem Cells in Disease Modeling and Drug Discovery for Myotonic Dystrophy Type 1

Myotonic dystrophy type 1 (DM1) is a progressive multisystemic disease caused by the expansion of a CTG repeat tract within the 3′ untranslated region (3′ UTR) of the dystrophia myotonica protein kinase gene (<i>DMPK</i>). Although DM1 is considered to be the most frequent myopathy of ge...

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Bibliographic Details
Main Authors: Noémie Bérenger-Currias, Cécile Martinat, Sandrine Baghdoyan
Format: Article
Language:English
Published: MDPI AG 2023-02-01
Series:Cells
Subjects:
myotonic dystrophy type 1
human pluripotent stem cells
disease modeling
drug screening
Online Access:https://www.mdpi.com/2073-4409/12/4/571

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