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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Bibliographic Details
Main Authors: Katherine G. English, Andrea L. Reid, Adrienne Samani, Gerald J. F. Coulis, S. Armando Villalta, Christopher J. Walker, Sharon Tamir, Matthew S. Alexander
Format: Article
Language:English
Published: MDPI AG 2022-09-01
Series:Biomedicines
Subjects:
Online Access:https://www.mdpi.com/2227-9059/10/10/2400