Development of in vivo gene therapy using exosomes loading immunomodulatory molecules

2020 Fiscal Year Final Research Report

• Project/Area Number: 18H02562
• Research Category: Grant-in-Aid for Scientific Research (B)
• Allocation Type: Single-year Grants
• Section: 一般
• Review Section: Basic Section 47020:Pharmaceutical analytical chemistry and physicochemistry-related
• Research Institution: Kyoto University
• Principal Investigator: Takakura Yoshinobu 京都大学, 薬学研究科, 教授 (30171432)
• Co-Investigator(Kenkyū-buntansha): 高橋 有己 京都大学, 薬学研究科, 准教授 (00547870)
• Project Period (FY): 2018-04-01 – 2021-03-31
• Keywords: エキソソーム / ルシフェラーゼ / 免疫療法

Outline of Final Research Achievements

In this study, we analyzed the behavior of endogenous exosomes and developed a method to attach immunoregulatory molecules to exosomes in order to develop in vivo gene immunotherapy using endogenous exosomes produced in the body. We focused on blood exosomes as endogenous exosomes, and succeeded in establishing a method to collect and label them. We selected NBD peptide, an NF-κB inhibitory peptide, as an immunoregulatory molecule, and succeeded in developing an efficient method for loading NBD peptide into exosomes. The NBD peptide-loaded exosomes showed a high anti-inflammatory effect. In conclusion, we have succeeded in establishing the basis for in vivo gene immunotherapy using endogenous exosomes.

Free Research Field

生物薬剤学

Academic Significance and Societal Importance of the Research Achievements

細胞から産生される粒子径100nm程度の膜小胞であるエキソソームは、細胞間で物質輸送を行う輸送担体であり、その生理機能の解明とそれを利用した治療法の開発が期待される。本研究では、体内におけるエキソソームの挙動の解明と治療用分子の搭載方法の確立に成功したが、これはエキソソームの生理機能の解明を目的とした研究並びに治療法の開発において重要な知見を提供するものである。