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

Dystrophinopathies are caused by mutations in the DMD 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 exon skipping convert...

Descripción completa

Detalles Bibliográficos
Autores principales: Anwar, Saeed, He, Merry, Lim, Kenji Rowel Q., Maruyama, Rika, Yokota, Toshifumi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7830903/
https://www.ncbi.nlm.nih.gov/pubmed/33466756
http://dx.doi.org/10.3390/jpm11010046

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7830903/
https://www.ncbi.nlm.nih.gov/pubmed/33466756
http://dx.doi.org/10.3390/jpm11010046

Ejemplares similares