Report Summary

Toxicology and Biodistribution Study of AAV-Mediated Gene Therapy for Muscular Dystrophy

Study Summary

Overall Study Design 
The objective of this study was to determine the potential toxicity of adeno-associated virus serotype 1 expressing the 
human alpha sarcoglycan gene rAAV1-tMCK-h-alphaSG following a single intramuscular IM injection into 
alpha-Sarcoglycan Knockout mice 
The study consisted of 4 experimental arms the mice were injected with either Lactated Ringers vehicle control group or 
rAAV1-tMCK-h-alphaSG at a total dose of 3e8 vg 12 x 10e13 vgkg Each arm consisted of 30 mice which included 5 
males and 5 females at each sacrifice time point of either 21 60 or 90 days The primary safety endpoint of this study was 
the histopathologic examination and detection of vector genomes in the blood skeletal muscle of the quadriceps both 
injected and contra lateral TA muscle both injected and contra lateral lung heart liver lymph nodes jejunum pancreas 
diaphragm brain spleen kidney gonads and lumbar spinal cord Clinical assessments were also measured by CBC and 
serum chemistry analysis 
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