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Development of a gene therapy targeting the vestibular organs by local administration of adeno-associated virus in the inner ear.

Research Project

Project/Area Number 18K16858
Research Category

Grant-in-Aid for Early-Career Scientists

Allocation TypeMulti-year Fund
Review Section Basic Section 56050:Otorhinolaryngology-related
Research InstitutionJuntendo University

Principal Investigator

Hiroko Okada  順天堂大学, 医学部, 助教 (20433774)

Project Period (FY) 2018-04-01 – 2022-03-31
Project Status Completed (Fiscal Year 2021)
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)
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
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.

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。

Report

(5 results)
  • 2021 Annual Research Report   Final Research Report ( PDF )
  • 2020 Research-status Report
  • 2019 Research-status Report
  • 2018 Research-status Report
  • Research Products

    (8 results)

All 2021 2020 2019 2018

All Journal Article (5 results) (of which Int'l Joint Research: 1 results,  Peer Reviewed: 5 results,  Open Access: 3 results) Presentation (3 results)

  • [Journal Article] Combined Underwater Endoscopic and Microscopic Surgery for External Auditory Canal Cholesteatoma:A case report2021

    • Author(s)
      Anzai T,Takata Y,Hara S,Sonoda K,Ikeda K,Okada H,Matsumoto F
    • Journal Title

      Ear,Nose & Throat Journal

      Volume: 7 Issue: 7 Pages: 0-0

    • DOI

      10.1177/01455613211064011

    • Related Report
      2021 Annual Research Report
    • Peer Reviewed / Open Access
  • [Journal Article] Association Between Earwax-Determinant Genotypes and Acquired Middle Ear Cholesteatoma in a Japanese Population2021

    • Author(s)
      Hara Satoshi、Kusunoki Takeshi、Nakagawa Hiroshi、Toyoda Yu、Nojiri Shuko、Kamiya Kazusaku、Furukawa Masayuki、Takata Yusuke、Okada Hiroko、Anzai Takashi、Matsumoto Fumihiko、Ikeda Katsuhisa
    • Journal Title

      Otolaryngology Head and Neck Surgery

      Volume: - Issue: 1 Pages: 139-145

    • DOI

      10.1177/01945998211000374

    • Related Report
      2021 Annual Research Report 2020 Research-status Report
    • Peer Reviewed / Open Access / Int'l Joint Research
  • [Journal Article] Cholesteatoma Surgery With a Dehiscent High Jugular Bulb Treated With Surgery Assisted With Underwater Endoscopy: A Case Report.2021

    • Author(s)
      Takata Y, Anzai T, Hara S, Okada H, Matsumoto F, Ikeda K
    • Journal Title

      Ear Nose Throat J

      Volume: 29 Issue: 7 Pages: 0-0

    • DOI

      10.1177/01455613211009135

    • Related Report
      2021 Annual Research Report
    • Peer Reviewed / Open Access
  • [Journal Article] Postoperative Management of Eosinophilic Chronic Rhinosinusitis with Nasal Polyps: Impact of High-Dose Corticosteroid Nasal Spray.2019

    • Author(s)
      Ikeda K, Ito S, Hibiya R, Homma H, Ono N, Okada H, Kidokoro Y, Shiozawa A, Kusunoki T.
    • Journal Title

      Int Arch Otorhinolaryngol

      Volume: 23 Issue: 01 Pages: 101-103

    • DOI

      10.1055/s-0038-1668515

    • Related Report
      2018 Research-status Report
    • Peer Reviewed
  • [Journal Article] Parapharyngeal Abscesses Caused by Group G Streptococcus2018

    • Author(s)
      Tajima Shori、Anzai Takashi、Matsuoka Rina、Okada Hiroko、Ide Takuma、Fujimaki Mitsuhisa、Kaya Shota、Ito Shin、Ikeda Katsuhisa
    • Journal Title

      Case Reports in Otolaryngology

      Volume: 2018 Pages: 1-4

    • DOI

      10.1155/2018/7307290

    • Related Report
      2018 Research-status Report
    • Peer Reviewed
  • [Presentation] 耳垢腺癌の1症例2020

    • Author(s)
      岡田 弘子, 古川 正幸, 高田 雄介, 池田 勝久
    • Organizer
      第30回日本耳科学会
    • Related Report
      2020 Research-status Report
  • [Presentation] 先天性真珠腫の顕微鏡下耳科手術3症例2020

    • Author(s)
      高田 雄介, 安齋 崇, 原 聡, 岡田 弘子, 田島 勝利, 池田 勝久
    • Organizer
      第30回日本耳科学会
    • Related Report
      2020 Research-status Report
  • [Presentation] 慢性浸潤性真菌性鼻副鼻腔炎の血清学的抗原マーカーを用いた非侵襲的な早期診断2020

    • Author(s)
      池田 勝久, 大庭 亜由子, 伊藤 伸, 岡田 弘子, 安齋 崇
    • Organizer
      第59回日本鼻科学会
    • Related Report
      2020 Research-status Report

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Published: 2018-04-23   Modified: 2023-01-30  

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