| Project/Area Number |
20591264
|
|---|
| Research Category |
Grant-in-Aid for Scientific Research (C)
|
| Allocation Type | Single-year Grants |
|---|
| Section | 一般 |
|---|
| Research Field |
Pediatrics
|
|---|
| Research Institution | Tokyo Women's Medical University |
|---|
Principal Investigator |
UTSUGISAWA Taiju Tokyo Women's Medical University, 医学部, 助教 (30337133)
|
|---|
| Co-Investigator(Kenkyū-buntansha) |
KANNO Hitoshi 東京女子医科大学, 医学部, 准教授 (70221207)
藤井 寿一 東京女子医科大学, 医学部, 教授 (70107762)
|
|---|
| Co-Investigator(Renkei-kenkyūsha) |
FUJII Hisaichi 東京女子医科大学, 医学部, 教授 (70107762)
|
|---|
| Project Period (FY) |
2008 – 2010
|
| Project Status |
Completed (Fiscal Year 2010)
|
| Budget Amount *help |
¥4,680,000 (Direct Cost: ¥3,600,000、Indirect Cost: ¥1,080,000)
Fiscal Year 2010: ¥910,000 (Direct Cost: ¥700,000、Indirect Cost: ¥210,000)
Fiscal Year 2009: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
Fiscal Year 2008: ¥2,210,000 (Direct Cost: ¥1,700,000、Indirect Cost: ¥510,000)
|
|---|
| Keywords | 遺伝子治療 / PK異常症 / レンチウイルスベクター / ピルビン酸キナーゼ異常症 / siRNA |
|---|
| Research Abstract |
To establish effective hematopietic stem cell targeted gene therapy for pyruvate kinase (PK) deficiency, we generated a lentiviral vector based on a novel strategy. The lentiviral vector is capable of dual expression of short interfering RNA (siRNA) against mutated mRNA and siRNA resistant wild type PK mRNA, resulted in substitution of abnormal PK for the normal enzyme in vivo.
|
