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...
Main Authors: | , , |
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Format: | Article |
Language: | English |
Published: |
MDPI AG
2023-02-01
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Series: | Cells |
Subjects: | |
Online Access: | https://www.mdpi.com/2073-4409/12/4/571 |