A First Step in Viral Gene Therapy for Muscular Dystrophy
โ Scribed by Michio Hirano
- Book ID
- 107545736
- Publisher
- Springer
- Year
- 2010
- Tongue
- English
- Weight
- 72 KB
- Volume
- 10
- Category
- Article
- ISSN
- 1528-4042
No coin nor oath required. For personal study only.
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## Abstract Duchenne's muscular dystrophy (DMD), which affects 1/3500 live male births, involves a progressive degeneration of skeletal and cardiac muscle, leading to early death. The protein dystrophin is lacking in DMD and present, but defective, in the allelic, less severe, Becker muscular dystr
## Abstract This commentary highlights the promising results of recent studies in animal models of Duchenne muscular dystrophy and amyotrophic lateral sclerosis that have clearly demonstrated the potential of gene therapy for tackling these diseases. In the absence of effective drugs or other treat