Development of a gene therapy targeting the vestibular organs by local administration of adeno-associated virus in the inner ear.
| Project/Area Number |
18K16858
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| Research Category |
Grant-in-Aid for Early-Career Scientists
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| Allocation Type | Multi-year Fund |
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| Review Section |
Basic Section 56050:Otorhinolaryngology-related
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| Research Institution | Juntendo University |
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Principal Investigator |
Hiroko Okada 順天堂大学, 医学部, 助教 (20433774)
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| Project Period (FY) |
2018-04-01 – 2022-03-31
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| Project Status |
Completed (Fiscal Year 2021)
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| Budget Amount *help |
¥4,160,000 (Direct Cost: ¥3,200,000、Indirect Cost: ¥960,000)
Fiscal Year 2021: ¥390,000 (Direct Cost: ¥300,000、Indirect Cost: ¥90,000)
Fiscal Year 2020: ¥390,000 (Direct Cost: ¥300,000、Indirect Cost: ¥90,000)
Fiscal Year 2019: ¥1,690,000 (Direct Cost: ¥1,300,000、Indirect Cost: ¥390,000)
Fiscal Year 2018: ¥1,690,000 (Direct Cost: ¥1,300,000、Indirect Cost: ¥390,000)
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| Keywords | 遺伝性難聴 / コネキシン26 / アデノ随伴ウィルス / 遺伝子難聴 / アデノ随伴ウイルス / 遺伝子治療 / 前庭 |
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| Outline of Final Research Achievements |
The Gjb2 gene, which encodes connexin 26 (CX26), a gap junction protein, is the most frequent causative gene in hereditary hearing loss. CX26 is expressed in the outer cochlear wall fiber cells and Corticoid supporting cells, but is also expressed in the vestibular organs. In this project, we will develop a method of administering Gjb2 gene therapy vectors targeting both vestibular organs and the cochlea, and analyze the function of vestibular organs after gene therapy to optimize gene transfer methods using adeno-associated viruses (AAV) into the vestibule. To administrate the AAV to the vestibular epithelial cells, we made a small hole in the semicircular canal, injected virus solution via microtube.After a certain period of time, AAV infections of the vestibular and semicircular canal areas were confirmed.
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| Academic Significance and Societal Importance of the Research Achievements |
先天性高度難聴の発症率は1,000人に1人の割合であり、その約半数は遺伝子の関与があるといわれている。遺伝性難聴の中でも最も頻度が高い原因遺伝子は、 ギャップ結合蛋白であるコネキシン26をコードしているGjb2遺伝子である。先天性高度難聴児は幼少児期に末梢平衡機能にも障害を伴う頻度が極めて高いことが 知られており著しく患者のQOLが低下するためその治療法の確立が強く求められている。前庭器官と蝸牛を共に標的としたGjb2遺伝子治療ベクターの投与法の開発は低侵襲な治療法であり、その治療法の確立は学術的および社会的意義は高いといえる。
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Report
(5 results)
Research Products
(8 results)
