Basic research for the establishment of human embryonic gene therapy

2022 Fiscal Year Final Research Report

• Project/Area Number: 20K09742
• Research Category: Grant-in-Aid for Scientific Research (C)
• Allocation Type: Multi-year Fund
• Section: 一般
• Review Section: Basic Section 56050:Otorhinolaryngology-related
• Research Institution: Osaka Metropolitan University (2022); The Tazuke Kofukai (2020-2021)
• Principal Investigator: Miwa Toru 大阪公立大学, 大学院医学研究科, 講師 (70535591)
• Project Period (FY): 2020-04-01 – 2023-03-31
• Keywords: 遺伝子治療 / Slc26a4 / Connexin30

Outline of Final Research Achievements

We generated the Slc26a4-PA-Tet system vector and transferred the PA-Tet system vector by electroporating Slc26a4-deficient mouse inne ear (embryonic day 11.5). We constructed a model mouse in which the temporal expression control. The tranduced location was confined to the endolymphatic sac, and hearing of this mice was rescued.

Free Research Field

耳鼻咽喉科

Academic Significance and Societal Importance of the Research Achievements

PA-Tet system制御ベクターに組織特異的なプロモーターを組み込み、青色光照射とドキシサイクリン経口投与による組織特異的遺伝子発現が可能なモデルマウスを作製し、異所性過剰発現実験を通して、遺伝子発現部位と内耳の機能・形態異常発現の関係について多角的検討を行うことができた。