2016 Fiscal Year Final Research Report
Gene therapy for neurodegenerative diseases using modified AAV vectors.
| Project/Area Number |
26293213
|
|---|
| Research Category |
Grant-in-Aid for Scientific Research (B)
|
| Allocation Type | Partial Multi-year Fund |
|---|
| Section | 一般 |
|---|
| Research Field |
Neurology
|
|---|
| Research Institution | Jichi Medical University |
|---|
Principal Investigator |
|
|---|
| Project Period (FY) |
2014-04-01 – 2017-03-31
|
| Keywords | 遺伝子治療 / 脊髄小脳失調症 / パーキンソン病 / AAVベクター |
|---|
| 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.
|
| Free Research Field |
神経内科学
|
