| Project/Area Number |
16K15140
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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) |
2016-04-01 – 2017-03-31
|
| Project Status |
Completed (Fiscal Year 2016)
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| Budget Amount *help |
¥3,640,000 (Direct Cost: ¥2,800,000、Indirect Cost: ¥840,000)
Fiscal Year 2016: ¥3,640,000 (Direct Cost: ¥2,800,000、Indirect Cost: ¥840,000)
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| Keywords | 核酸医薬 / アンチセンス核酸医薬 / 2本鎖核酸医薬 / 核酸 |
|---|
| Outline of Final Research Achievements |
Here we developed a new class of RNaseH independent therapeutics oligonucleotide without a ligand conjugation. We assessed in vitro and in vivo efficacy of double-stranded SSO (splicing switching oligonucleotide) targeting dystrophin, which is causative gene of Duchenne muscular dystrophy (DMD). We achieved exon skipping of the dystrophin mRNA by double-stranded SSO in vitro and in vivo.
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