2010 Fiscal Year Final Research Report
A novel gene therapy for pyruvate kinase deficiency.
| Project/Area Number |
20591264
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| Research Category |
Grant-in-Aid for Scientific Research (C)
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| Allocation Type | Single-year Grants |
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| Section | 一般 |
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| Research Field |
Pediatrics
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| Research Institution | Tokyo Women's Medical University |
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Principal Investigator |
UTSUGISAWA Taiju Tokyo Women's Medical University, 医学部, 助教 (30337133)
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| Co-Investigator(Kenkyū-buntansha) |
KANNO Hitoshi 東京女子医科大学, 医学部, 准教授 (70221207)
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| Co-Investigator(Renkei-kenkyūsha) |
FUJII Hisaichi 東京女子医科大学, 医学部, 教授 (70107762)
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| Project Period (FY) |
2008 – 2010
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| Keywords | 遺伝子治療 / PK異常症 / レンチウイルスベクター |
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| 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.
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