Development of a novel RNAi medicine based on shRNA expression mini-plasmid

2014 Fiscal Year Final Research Report

• Project/Area Number: 24390027
• Research Category: Grant-in-Aid for Scientific Research (B)
• Allocation Type: Partial Multi-year Fund
• Section: 一般
• Research Field: Drug development chemistry
• Research Institution: The University of Tokushima
• Principal Investigator: MINAKAWA Noriaki 徳島大学, ヘルスバイオサイエンス研究部, 教授 (40209820)
• Co-Investigator(Kenkyū-buntansha): ISHIDA Tstsuhiro 徳島大学, 大学院ヘルスバイオサイエンス研究部, 教授 (50325271)
• Project Period (FY): 2012-04-01 – 2015-03-31
• Keywords: RNAi創薬 / 4'-チオDNA / PCR / 非天然塩基対 / クリックケミストリー / 免疫応答

Outline of Final Research Achievements

In this study, I aim at a development of novel RNAi medicine based on shRNA expression mini-gene. As a result, I succeeded to prepare the mini-gene via PCR amplification using 2’-deoxy-4’-thionucleoside triphosphates (dSNTPs). In addition, I also developed a practical method to cyclize the resulting mini-gene.
The mini-gene showed potent RNAi activity both in vitro and in vivo. In addition, innate immune responses such as type-I INF and inflammatory cytokines were hardly induced by administration of the mini-gene in vivo.

Free Research Field

核酸創薬