2009 Fiscal Year Final Research Report
In Vivo Application of an RNAi Strategy for the Selective Suppression of a Mutant Allele
| Project/Area Number |
20790610
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| Research Category |
Grant-in-Aid for Young Scientists (B)
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| Allocation Type | Single-year Grants |
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| Research Field |
Neurology
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| Research Institution | Tokyo Medical and Dental University |
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Principal Investigator |
KUBODERA Takayuki Tokyo Medical and Dental University, 医学部・附属病院, 医員 (40376801)
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| Project Period (FY) |
2008 – 2009
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| Keywords | siRNA / 遺伝子治療 / トランスジェニックマウス / ALS / SOD1 / AAV |
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| Research Abstract |
We previously proposed a strategy for selective suppression of mutant allele. We succeeded in mutant gene suppression in the target organ of mice, using in vivo administered adeno-associated viral vectors. Our method may prove useful for siRNA-based gene therapy for inherited diseases.
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