| Budget Amount *help |
¥16,510,000 (Direct Cost: ¥12,700,000、Indirect Cost: ¥3,810,000)
Fiscal Year 2016: ¥5,590,000 (Direct Cost: ¥4,300,000、Indirect Cost: ¥1,290,000)
Fiscal Year 2015: ¥5,590,000 (Direct Cost: ¥4,300,000、Indirect Cost: ¥1,290,000)
Fiscal Year 2014: ¥5,330,000 (Direct Cost: ¥4,100,000、Indirect Cost: ¥1,230,000)
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| Outline of Final Research Achievements |
Using modified adeno-associated virus (AAV) vectors that can delivery therapeutic genes to neurons in broad areas of the central nervous system, we have developed gene therapy for neurodegenerative diseases. Spinocerebellar ataxia type 6 (SCA6) is caused by a polyglutamine repeat expansion within a second CACNA1A gene product, α1ACT. As a potential therapy, the complete silencing of CACNA1A gene expression would be lethal, although the selective elimination of α1ACT protein could be a viable strategy. AAV vector-mediated delivery of miR-3191-5p into the ventricles protected from the Purkinje cell degeneration and ataxia in a mouse model that was made by intraventricular infusion of AAV vector expressing expanded polyglutamine repeats in the α1ACT gene. We also confirmed that transgenes were expressed even after 15 years in a monkey model of Parkinson disease.
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