| Project/Area Number |
24791060
|
|---|
| Research Category |
Grant-in-Aid for Young Scientists (B)
|
| Allocation Type | Multi-year Fund |
|---|
| Research Field |
Pediatrics
|
|---|
| Research Institution | Kobe University |
|---|
Principal Investigator |
NAKAGAWA Taku 神戸大学, 医学部附属病院, 助教 (40457073)
|
|---|
| Co-Investigator(Renkei-kenkyūsha) |
NISHIO Hisahide 神戸大学, 大学院医学研究科, 教授 (80189258)
|
|---|
| Project Period (FY) |
2012-04-01 – 2015-03-31
|
| Project Status |
Completed (Fiscal Year 2014)
|
| Budget Amount *help |
¥4,160,000 (Direct Cost: ¥3,200,000、Indirect Cost: ¥960,000)
Fiscal Year 2013: ¥1,820,000 (Direct Cost: ¥1,400,000、Indirect Cost: ¥420,000)
Fiscal Year 2012: ¥2,340,000 (Direct Cost: ¥1,800,000、Indirect Cost: ¥540,000)
|
|---|
| Keywords | spinal muscular atrophy / SMA / splicing / SMN2 |
|---|
| Outline of Final Research Achievements |
Spinal muscular atrophy is a fatal congenital neuromuscular disease caused by SMN1 gene mutation. Effective treatment has not established yet. As a strategy of the treatment, modification for SMN2 splicing is thought to be a promising approach. We previously discovered compound A is effective to modify the SMN2 splicing using mini gene system. Subsequently, we tried to search for similar compound that would modify the SMN2 splicing safely. However, we didn't find the effective compounds for SMN2 splicing correction during the study period.
|
