Next-Generation SINE Compound KPT−8602 Ameliorates Dystrophic Pathology in Zebrafish and Mouse Models of DMD

Duchenne muscular dystrophy (DMD) is a progressive, X-linked childhood neuromuscular disorder that results from loss-of-function mutations in the DYSTROPHIN gene. DMD patients exhibit muscle necrosis, cardiomyopathy, respiratory failure, and loss of ambulation. One of the major driving forces of DMD...

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Detalles Bibliográficos
Autores principales: English, Katherine G., Reid, Andrea L., Samani, Adrienne, Coulis, Gerald J. F., Villalta, S. Armando, Walker, Christopher J., Tamir, Sharon, Alexander, Matthew S.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9598711/
https://www.ncbi.nlm.nih.gov/pubmed/36289662
http://dx.doi.org/10.3390/biomedicines10102400

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