AAV9-NPC1 significantly ameliorates Purkinje cell death and behavioral abnormalities in mouse NPC disease
Niemann-Pick type C (NPC) disease is a fatal inherited neurodegenerative disorder caused
by loss-of-function mutations in the NPC1 or NPC2 gene. There is no effective way to treat
NPC disease. In this study, we used adeno-associated virus (AAV) serotype 9 (AAV9) to
deliver a functional NPC1 gene systemically into NPC1−/− mice at postnatal day 4. One
single AAV9-NPC1 injection resulted in robust NPC1 expression in various tissues,
including brain, heart, and lung. Strikingly, AAV9-mediated NPC1 delivery significantly …
by loss-of-function mutations in the NPC1 or NPC2 gene. There is no effective way to treat
NPC disease. In this study, we used adeno-associated virus (AAV) serotype 9 (AAV9) to
deliver a functional NPC1 gene systemically into NPC1−/− mice at postnatal day 4. One
single AAV9-NPC1 injection resulted in robust NPC1 expression in various tissues,
including brain, heart, and lung. Strikingly, AAV9-mediated NPC1 delivery significantly …
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