• Search Research Projects
  • Search Researchers
  • How to Use
  1. Back to project page

2020 Fiscal Year Final Research Report

Development of a non-invasive and area selective gene transfer method for the primate brain

Research Project

  • PDF
Project/Area Number 19K22470
Research Category

Grant-in-Aid for Challenging Research (Exploratory)

Allocation TypeMulti-year Fund
Review Section Medium-sized Section 46:Neuroscience and related fields
Research InstitutionKyoto University

Principal Investigator

Inoue Kenichi  京都大学, 霊長類研究所, 助教 (90455395)

Project Period (FY) 2019-06-28 – 2021-03-31
Keywords神経科学 / 脳・神経 / バイオテクノロジー / ウイルスベクター / 霊長類
Outline of Final Research Achievements

This study investigated the feasibility of noninvasive gene transfer to the target brain region in monkeys, a model animal closely related to humans. We first developed a capsid-modified adeno-associated virus vector that enables gene transfer to neurons throughout the brain by intravenous administration to newborn monkeys. Moreover, in collaboration with HM CINAC, Spain, we have achieved gene transfer to specific brain regions in adult macaque monkeys by the transient opening of blood-brain barrier (BBB) using transcranial focused ultrasound (tFUS), although there is room for improvement in efficiency. In this study, we further applied cell type-specific promoters to these systems and succeeded in achieving cell type-specific gene expression.

Free Research Field

神経科学、ウイルス学

Academic Significance and Societal Importance of the Research Achievements

発生工学を利用せず、ウイルスベクターによって霊長類における領域特異的、あるいは全脳的なニューロンへの遺伝子導入を実現するという本研究の成果は、選択的な遺伝子操作系など幅広い応用が可能な先端的技術となると考えられ、遺伝子改変霊長類疾患モデル動物の作出という観点から、精神・神経疾患の病態解明と治療法の開発に極めて大きく寄与すると考える。また、非侵襲的に目的とする領野に選択的な遺伝子導入を可能にする本研究の成果は、遺伝子治療法の開発の上で重要な知見を与えるものであり、特に遺伝子疾患の有効な治療法の開発研究に大きく貢献できる。

URL: 

Published: 2022-01-27  

Information User Guide FAQ News Terms of Use Attribution of KAKENHI

Powered by NII kakenhi