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Report Summary

Preclinical Study of Adeno-Associated Virus Mediated Gene Therapy for Pompe Disease (Study ID# GSDII-001)


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 acid alpha-Glucosidase hGAAdriven by a cytomegalovirus CMVpromoter rAAV1-p432-hGAA following 
muscular injection in GAA 1296neo6neo mice 
The study consisted of 4 experimental groups The animals were injected intramuscularly within the right quadriceps at one 
site with either Lactated Ringers vehicle control group or rAAV1-p432-hGAA at a total dose of 3 x 10e9 3 x 10e10 or 3 x 
10e11 vg Each arm consisted of 30 animals 
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