2012 Fiscal Year Final Research Report
Development of gene therapy using novel double-stranded antisense oligonucleotides
| Project/Area Number |
24659258
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| Research Category |
Grant-in-Aid for Challenging Exploratory Research
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| Allocation Type | Multi-year Fund |
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| Research Field |
Applied pharmacology
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| Research Institution | Tokyo Medical and Dental University |
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Principal Investigator |
NISHINA Kazutaka 東京医科歯科大学, 大学院・医歯学総合研究科, メディカルフェロー (60622529)
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| Project Period (FY) |
2012
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| Keywords | 遺伝子診断 / 治療 |
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| Research Abstract |
Here we developed a new class of exceptionally potent antisense oligonucleotides (ASOs) that are hybridized with complementary RNA (cRNA) and conjugated with a ligand. At first, we optimized the chemical modifications of cRNA. Secondly, tocopherol-conjugated ASO (Toc-dsASO) reduced multiple target sequence including mouse apolipoprotein B and human transthyretin. Finally, we showed the liver specific delivery of Toc-dsASO using IVIS imaging system.
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