Mutation-Based Therapeutic Strategies for Duchenne Muscular Dystrophy: From Genetic Diagnosis to Therapy

Mutation-Based Therapeutic Strategies for Duchenne Muscular Dystrophy: From Genetic Diagnosis to Therapy

Duchenne and Becker muscular dystrophy (DMD/BMD) are X-linked muscle disorders caused by mutations of the DMD gene, which encodes the subsarcolemmal protein dystrophin. In DMD, dystrophin is not expressed due to a disruption in the reading frame of the DMD gene, resulting in a severe phenotype. Beck...

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Bibliographic Details
Main Author: Akinori Nakamura
Format: Article
Language:English
Published: MDPI AG 2019-03-01
Series:Journal of Personalized Medicine
Subjects:
Duchenne/Becker muscular dystrophy (DMD/BMD)
DMD gene
dystrophin
molecular diagnosis
DMD model animal
gene therapy
readthrough therapy
exon skipping therapy
antisense oligonucleotide
Online Access:http://www.mdpi.com/2075-4426/9/1/16

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http://www.mdpi.com/2075-4426/9/1/16

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