Development of functional lipid nanoparticles for RNA interference-based therapy

2018 Fiscal Year Final Research Report

• Project/Area Number: 16K08201
• Research Category: Grant-in-Aid for Scientific Research (C)
• Allocation Type: Multi-year Fund
• Section: 一般
• Research Field: Physical pharmacy
• Research Institution: University of Shizuoka
• Principal Investigator: Tomohiro Asai 静岡県立大学, 薬学部, 教授 (00381731)
• Project Period (FY): 2016-04-01 – 2019-03-31
• Keywords: 脂質ナノ粒子 / RNA干渉 / オートファジー / siRNA / verteporfin / DDS

Outline of Final Research Achievements

Small interfering RNA (siRNA) capable of inducing gene silencing is a promising drug candidate to address unmet medical needs. However, siRNA is difficult to induce gene silencing without an appropriate delivery vehicle. The establishment of effective delivery systems is one of the most important tasks to develop RNA interference (RNAi)-based drugs. Functional lipid nanoparticles are attractive and promising vectors for RNAi therapy. In the present study, we developed novel lipid nanoparticles containing a possible RNAi enhancer for cancer treatment. Our findings provide new insights into the design of effective lipid nanoparticles for RNAi therapy.

Free Research Field

薬物送達学

Academic Significance and Societal Importance of the Research Achievements

RNA干渉薬をアプタマー医薬や抗体医薬と比較すると、細胞内タンパク質が創薬ターゲットになるという利点が大きく、DDSの技術革新次第では数多くの画期的医薬品が創出される可能性がある。つまり、RNAを安全に患部に運搬するシステムが確立されれば、アンドラッガブル問題の解消へと繋がり、医療の進歩に大きく貢献できる。本研究の成果は、RNA干渉薬開発に必要なDDSについて重要な基礎的知見を与えるものであり、その学術的意義は大きい。