Antisense and Gene Therapy Options for Duchenne Muscular Dystrophy Arising from Mutations in the N-Terminal Hotspot

Duchenne muscular dystrophy (DMD) is a fatal genetic disease affecting children that is caused by a mutation in the gene encoding for dystrophin. In the absence of functional dystrophin, patients experience progressive muscle deterioration, leaving them wheelchair-bound by age 12 and with few patien...

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Detalles Bibliográficos
Autores principales: Wilton-Clark, Harry, Yokota, Toshifumi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8872079/
https://www.ncbi.nlm.nih.gov/pubmed/35205302
http://dx.doi.org/10.3390/genes13020257

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8872079/
https://www.ncbi.nlm.nih.gov/pubmed/35205302
http://dx.doi.org/10.3390/genes13020257

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