NeuroscienceLife Science AlliancePeer-reviewed
Optimization of systemic AAV9 gene therapy in Niemann-Pick disease type C1 mice
Avani V. Mylvara, Alana L. Gibson, Tansy Gu…·William J. Pavan
This study evaluates the efficacy of systemic AAV9-hNPC1 gene therapy in NPC1 mouse models, finding that higher doses and earlier treatment improve survival and slow disease progression.
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