In vivo suppression of apoptosis signaling using mRNA therapeutics for the treatment of ischemic diseases

2017 Fiscal Year Final Research Report

• Project/Area Number: 15K20962
• Research Category: Grant-in-Aid for Young Scientists (B)
• Allocation Type: Multi-year Fund
• Research Field: Biomedical engineering/Biomaterial science and engineering; Applied pharmacology
• Research Institution: The University of Tokyo
• Principal Investigator: Uchida Satoshi 東京大学, 大学院工学系研究科(工学部), 特任助教 (20710726)
• Project Period (FY): 2015-04-01 – 2018-03-31
• Keywords: mRNA医薬 / 高分子ミセル / 遺伝子治療 / アポトーシス

Outline of Final Research Achievements

Introduction of pro-survival gene is an attractive strategy for treatment of ischemic diseases, and mRNA therapeutics is a safe and promising methodology for this purpose. While mRNA is subjected to nuclease degradation soon after in vivo delivery, we successfully solved this issue by encapsulating mRNA into biocompatible polyplex micelles. Then, we attempted treatment of hind limb ischemia by delivering mRNA encoding myr-Akt, a pro-survival factor, using polyplex micelles. The treatment resulted in significant recovery of blood flow in mouse hind limb. The mRNA-based pro-survival factor introduction is a promising strategy for the treatment of various diseases accompanying enhanced cell death, including ischemic diseases.

Free Research Field

遺伝子治療、薬物送達システム