Development of novel triple-stranded antisense oligonucleotides
| Project/Area Number |
24659044
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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 |
Drug development chemistry
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| Research Institution | Tokyo Medical and Dental University |
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Principal Investigator |
YOKOTA Takanori 東京医科歯科大学, 大学院・医歯学総合研究科, 教授 (90231688)
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| Project Period (FY) |
2012
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| Project Status |
Completed (Fiscal Year 2012)
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| Budget Amount *help |
¥3,900,000 (Direct Cost: ¥3,000,000、Indirect Cost: ¥900,000)
Fiscal Year 2012: ¥3,900,000 (Direct Cost: ¥3,000,000、Indirect Cost: ¥900,000)
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| Keywords | 医薬分子設計 / アンチセンス / 核酸医薬 / デリバリー / 後根神経節細胞 / ELISA / RT-PCR / PNA / ホスホロチオエート結合修飾 |
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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) strand and peptide-conjugated PNA strand. At first, we optimized the chemical modifications and length of the strands of the triple-stranded ASO (tsASO). Secondly, peptide-conjugated tsASO reduced mouse TRPV1 gene in dorsal root ganglia (DRG). Finally, we showed the delivery of DRG-delivered peptide-conjugated tsASO using confocal imaging and quantitative RT-PCR by systemic administration.
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Report
(2 results)
Research Products
(3 results)
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[Journal Article] Intrathecal shRNA-AAV9 inhibits target protein expression in the spinal cord and dorsal root ganglia of adult mice.2012
Author(s)
Hirai T, Enomoto M, Machida A, Yamamoto M, Kuwahara H, Tajiri M, Hirai Y, Sotome S, Mizusawa H, Shinomiya K, Okawa A, Yokota T.
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Journal Title
Hum Gene Ther Methods
Volume: 23
Issue: 2
Pages: 119-27
DOI
Related Report
Peer Reviewed
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