Development of therapeutic oligonucleotides without ligand conjugation.

2016 Fiscal Year Final Research Report

• Project/Area Number: 16K15140
• Research Category: Grant-in-Aid for Challenging Exploratory Research
• Allocation Type: Multi-year Fund
• Research Field: Drug development chemistry
• Research Institution: Tokyo 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
• Keywords: 核酸医薬

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.

Free Research Field

遺伝子治療