Innovative Therapeutic Approaches for Duchenne Muscular Dystrophy

Innovative Therapeutic Approaches for Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is the most common childhood muscular dystrophy affecting ~1:5000 live male births. Following the identification of pathogenic variations in the dystrophin gene in 1986, the underlining genotype/phenotype correlations emerged and the role of the dystrophin protein w...

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Bibliographic Details
Main Authors: Fernanda Fortunato, Rachele Rossi, Maria Sofia Falzarano, Alessandra Ferlini
Format: Article
Language:English
Published: MDPI AG 2021-02-01
Series:Journal of Clinical Medicine
Subjects:
Duchenne muscular dystrophy
dystrophin restoration
antisense oligonucleotide chemistry
exon-skipping
stop codon reversion
gene therapy
Online Access:https://www.mdpi.com/2077-0383/10/4/820

Internet

https://www.mdpi.com/2077-0383/10/4/820

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