A Genotype-Phenotype Correlation Study of Exon Skip-Equivalent In-Frame Deletions and Exon Skip-Amenable Out-of-Frame Deletions across the <i>DMD</i> Gene to Simulate the Effects of Exon-Skipping Therapies: A Meta-Analysis

A Genotype-Phenotype Correlation Study of Exon Skip-Equivalent In-Frame Deletions and Exon Skip-Amenable Out-of-Frame Deletions across the <i>DMD</i> Gene to Simulate the Effects of Exon-Skipping Therapies: A Meta-Analysis

Dystrophinopathies are caused by mutations in the <i>DMD</i> gene. Out-of-frame deletions represent most mutational events in severe Duchenne muscular dystrophy (DMD), while in-frame deletions typically lead to milder Becker muscular dystrophy (BMD). Antisense oligonucleotide-mediated ex...

Full description

Bibliographic Details
Main Authors: Saeed Anwar, Merry He, Kenji Rowel Q. Lim, Rika Maruyama, Toshifumi Yokota
Format: Article
Language:English
Published: MDPI AG 2021-01-01
Series:Journal of Personalized Medicine
Subjects:
dystrophinopathy
duchenne muscular dystrophy (DMD)
becker muscular dystrophy (BMD)
dystrophin
reading frame rule
exon skipping
Online Access:https://www.mdpi.com/2075-4426/11/1/46

Internet

https://www.mdpi.com/2075-4426/11/1/46

Similar Items