Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy

Duchenne muscular dystrophy (DMD) is a progressive muscle-wasting disorder. It is caused by loss-of-function mutations in the dystrophin gene. Currently, there is no cure. A highly promising therapeutic strategy is to replace or repair the defective dystrophin gene by gene therapy. Numerous animal m...

Full description

Saved in:
Bibliographic Details
Published in:Disease models & mechanisms 2015-03, Vol.8 (3), p.195-213
Main Authors: McGreevy, Joe W., Hakim, Chady H., McIntosh, Mark A., Duan, Dongsheng
Format: Article
Language:English
Subjects:
Amino acids
Animal model
Animals
Binding sites
Canine DMD
Cardiomyopathies - therapy
Disease Models, Animal
Duchenne muscular dystrophy
Dystrophin
Dystrophin - deficiency
Dystrophin - metabolism
Extracellular matrix
Gene therapy
Genetic Therapy
Genomes
Glycoproteins
Lipids
Medical screening
Muscular dystrophy
Muscular Dystrophy, Duchenne - therapy
Mutation
Neurons - pathology
Nitric oxide
Plasmids
Proteins
Review
Vectors (Biology)
Citations: Items that this one cites
Items that cite this one
Online Access:Get full text
Tags: Add Tag
No Tags, Be the first to tag this record!