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Report Summary

Systemic Gene Transfer of a Hexosaminidase Variant Using an scAAV9.47 Vector Corrects G M2 Gangliosidosis in Sandhoff Mice


Study Summary

Overall Study Design 
 
This study evaluates the therapeutic effects of systemic delivery of scAAV947HEXM vector in the neonatal SD mouse 
model We sought to examine the phenotypic and biochemical effects of a single vector injection The primary aim of this 
study was to determine whether the newly developed HexM enzyme which was derived from the human beta-
hexosaminidase alpha-subunit can function with the murine GM2AP and provide behavioral and survival benefit 
 
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