Microdystrophin Gene Addition Significantly Improves Muscle Functionality and Diaphragm Muscle Histopathology in a Fibrotic Mouse Model of Duchenne Muscular Dystrophy

Microdystrophin Gene Addition Significantly Improves Muscle Functionality and Diaphragm Muscle Histopathology in a Fibrotic Mouse Model of Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a rare neuromuscular disease affecting 1:5000 newborn males. No cure is currently available, but gene addition therapy, based on the adeno-associated viral (AAV) vector-mediated delivery of microdystrophin transgenes, is currently being tested in clinical trials....

Full description

Bibliographic Details
Main Authors: Viktorija Cernisova, Ngoc Lu-Nguyen, Jessica Trundle, Shan Herath, Alberto Malerba, Linda Popplewell
Format: Article
Language:English
Published: MDPI AG 2023-05-01
Series:International Journal of Molecular Sciences
Subjects:
Duchenne muscular dystrophy
muscle pathology
AAV8-microdystrophin
fibrosis
Online Access:https://www.mdpi.com/1422-0067/24/9/8174

Internet

https://www.mdpi.com/1422-0067/24/9/8174

Similar Items