Development of prodrug-type siRNA with long term activities

2016 Fiscal Year Final Research Report

• Project/Area Number: 15K21517
• Research Category: Grant-in-Aid for Young Scientists (B)
• Allocation Type: Multi-year Fund
• Research Field: Physical pharmacy; Chemical pharmacy
• Research Institution: Osaka University of Pharmaceutical Sciences
• Principal Investigator: Hayashi Junsuke 大阪薬科大学, 薬学部, 助手(移行) (30740295)
• Project Period (FY): 2015-04-01 – 2017-03-31
• Keywords: 核酸医薬 / siRNA / プロドラッグ / ジスルフィド

Outline of Final Research Achievements

We have developed novel prodrug-type siRNAs containing 2'-O-methyldithiomethyl groups which responsive to intracellular reducing environment and transformable into natural 2'-OH RNA. The gene silencing effects of 2'-O-methyldithiomethyl (MDTM)-modified siRNAs were remarkably stronger than those of natural(2'-OH) siRNAs. Furthermore, the siRNA activities of 2'-O-MDTM-siRNA were enhanced by increasing of 2’-O-MDTM modifications.

Free Research Field

核酸化学