Key result
Intravenous delivery of an nNOS-binding micro-dystrophin vector restored dystrophin expression, reduced fibrosis and inflammation, and improved muscle function in DBA/2J-mdx mice.
Why the study?
Does a nNOS-binding micro-dystrophin vector improve dystrophic phenotype and cardiac function in the DBA/2J-mdx mouse model of Duchenne muscular dystrophy?
Population
10-week-old DBA/2J-mdx mice (severe model of Duchenne muscular dystrophy) and control DBA/2J mice
Comparison
Neuronal nitric oxide synthase-binding… vs Control DBA/2J mice
Design
Preclinical
Follow-up
Evaluated at 6 months of age
Authors
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Supports nNOS-binding micro-dystrophin for DMD skeletal muscle; leaves cardiac effects open in this unsuitable model.
Does a nNOS-binding micro-dystrophin vector improve dystrophic phenotype and cardiac function in the DBA/2J-mdx mouse model of Duchenne muscular dystrophy?
A micro-dystrophin gene vector rescues skeletal muscle phenotype in a severe Duchenne muscular dystrophy mouse model, but the DBA/2J-mdx model is unsuitable for evaluating Duchenne cardiomyopathy due to baseline cardiac defects in control mice.
Hakim et al. (2017) studied Duchenne muscular dystrophy. neuronal nitric oxide synthase (nNOS)-binding micro-dystrophin vector vs. control DBA/2J mice was evaluated on Disease rescue (muscle histology and function). Intravenous delivery of an nNOS-binding micro-dystrophin vector restored dystrophin expression, reduced fibrosis and inflammation, and improved muscle function in DBA/2J-mdx mice.
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