Molecular Therapies for Myotonic Dystrophy Type 1: From Small Drugs to Gene Editing

Molecular Therapies for Myotonic Dystrophy Type 1: From Small Drugs to Gene Editing

Myotonic dystrophy type 1 (DM1) is the most common muscular dystrophy affecting many different body tissues, predominantly skeletal and cardiac muscles and the central nervous system. The expansion of CTG repeats in the DM1 protein-kinase (<i>DMPK</i>) gene is the genetic cause of the di...

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Bibliographic Details
Main Authors: Mariapaola Izzo, Jonathan Battistini, Claudia Provenzano, Fabio Martelli, Beatrice Cardinali, Germana Falcone
Format: Article
Language:English
Published: MDPI AG 2022-04-01
Series:International Journal of Molecular Sciences
Subjects:
myotonic dystrophy
trinucleotide-expansion disease
DM1 mice
antisense oligonucleotides
molecular therapy
gene editing
Online Access:https://www.mdpi.com/1422-0067/23/9/4622

Internet

https://www.mdpi.com/1422-0067/23/9/4622

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