Long-term correction of Sandhoff disease following intravenous delivery of rAAV9 to mouse neonates.


G(M2) gangliosidoses are severe neurodegenerative disorders resulting from a deficiency in β-hexosaminidase A activity and lacking effective therapies. Using a Sandhoff disease (SD) mouse model (Hexb(-/-)) of the G(M2) gangliosidoses, we tested the potential of systemically delivered adeno-associated virus 9 (AAV9) expressing Hexb cDNA to correct the… (More)
DOI: 10.1038/mt.2014.240


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