Systemic Delivery of AAVB1-GAA Clears Glycogen and Prolongs Survival in a Mouse Model of Pompe Disease.
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DOI: 10.1089/hum.2018.016
Abstract: Pompe disease is an autosomal recessive glycogen storage disorder caused by deficiency of the lysosomal enzyme acid alpha-glucosidase (GAA). GAA deficiency results in systemic lysosomal glycogen accumulation and cellular disruption in muscle and the central… read more here.
Keywords: aavb1 gaa; gaa; glycogen; pompe disease ... See more keywords