Development of nanomachine for mitochondrial gene therapy targeted to cells derived from mitochondrial disease patients

2016 Fiscal Year Final Research Report

• Project/Area Number: 26282131
• Research Category: Grant-in-Aid for Scientific Research (B)
• Allocation Type: Partial Multi-year Fund
• Section: 一般
• Research Field: Biomedical engineering/Biomaterial science and engineering
• Research Institution: Hokkaido University
• Principal Investigator: YAMADA Yuma 北海道大学, 薬学研究科(研究院), 准教授 (60451431)
• Co-Investigator(Renkei-kenkyūsha): AKITA Hidetaka 千葉大学, 大学院薬学研究院, 教授 (80344472) ; HYODO Mamoru 愛知工業大学, 工学部, 講師 (30548186)
• Project Period (FY): 2014-04-01 – 2017-03-31
• Keywords: 薬物送達システム / ミトコンドリア

Outline of Final Research Achievements

In order to establish a therapeutic strategy targeting genes in mitochondria (Mt), we examined Mt gene delivery and mitochondrial exogenous gene expression in cells derived from Mt disease patients. To date, we have been able to develop a MITO-Porter, a nano carrier for Mt delivery. In this study, we report on the successful Mt gene delivery in disease cells that have a heteroplasmic mutation in mtDNA, using MITO-Porter system. In addition, we designed a pHSP-mtLuc (CGG) analog, an artificial Mt gene expression plasmid DNA vector that contains a promotor for Mt transcription and an artificial Mt genome with a reporter gene that records adjustments to the Mt codon system. Collectively, we succeeded in achieving Mt exogenous gene expression by the Mt delivery of a pHSP-mtLuc (CGG) analog using the developed MITO-Porter system. Our Mt exogenous gene expression carriers promises to be a useful approach to Mt gene therapy and research regarding Mt molecular biology.

Free Research Field

薬物送達学