Journal
JOURNAL OF VIROLOGY
Volume 74, Issue 3, Pages 1436-1442Publisher
AMER SOC MICROBIOLOGY
DOI: 10.1128/JVI.74.3.1436-1442.2000
Keywords
-
Categories
Funding
- NIAMS NIH HHS [R01AR45967-01, AR45925-01, R01 AR045967, P01 AR045925] Funding Source: Medline
Ask authors/readers for more resources
Limb girdle muscular dystrophy (LGMD) 2F is caused by mutations in the delta-sarcoglycan (SC) gene. Previously, we have shown successful application of a recombinant adeno-associated virus (AAV) vector for genetic and biochemical rescue in the Bio14.6 hamster, a homologous animal model for LGMD 2F (J. Li et al., Gene Ther, 6:74-82, 1999), In this report, we show efficient and long-term delta-SG expression accompanied by nearly complete recovery of physiological function deficits after a single-dose AAV vector injection into the tibialis anterior muscle of the dystrophic hamsters, AAV vector treatment led to more than 97% recovery in muscle strength for both the specific twitch forte and the specific tetanic force, when compared to the age-matched control, Vector treatment also prevented pathological muscle hypertrophy and resulted in normal muscle weight and size. Finally, vector-treated muscle showed substantial improvement of the histopathology, This is the first report of successful functional rescue of an entire muscle after AAV-mediated gene delivery. This report also demonstrates the feasibility of in vivo gene therapy for LGMD patients by using AAV vectors.
Authors
I am an author on this paper
Click your name to claim this paper and add it to your profile.
Reviews
Recommended
No Data Available