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Development of therapeutic oligonucleotides without ligand conjugation.

Research Project

Project/Area Number 16K15140
Research Category

Grant-in-Aid for Challenging Exploratory Research

Allocation TypeMulti-year Fund
Research Field Drug development chemistry
Research InstitutionTokyo Medical and Dental University

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)

Project Period (FY) 2016-04-01 – 2017-03-31
Project Status Completed (Fiscal Year 2016)
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)
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.

Report

(2 results)
  • 2016 Annual Research Report   Final Research Report ( PDF )

URL: 

Published: 2016-04-21   Modified: 2019-12-27  

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