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Modeling inner ear cilliopathy using patient derived iPS cells

Research Project

Project/Area Number 17K19733
Research Category

Grant-in-Aid for Challenging Research (Exploratory)

Allocation TypeMulti-year Fund
Research Field Surgery related to the biological and sensory functions and related fields
Research InstitutionKeio University

Principal Investigator

FUJIOKA Masato  慶應義塾大学, 医学部(信濃町), 講師 (70398626)

Co-Investigator(Kenkyū-buntansha) 小川 郁  慶應義塾大学, 医学部(信濃町), 教授 (00169179)
北尻 真一郎  信州大学, 学術研究院医学系(医学部附属病院), 講師 (00532970)
宇佐美 真一  信州大学, 学術研究院医学系, 教授 (10184996)
西尾 信哉  信州大学, 医学部, 特任講師 (70467166)
松永 達雄  独立行政法人国立病院機構(東京医療センター臨床研究センター), その他部局等, 部長 (90245580)
Project Period (FY) 2017-06-30 – 2020-03-31
Project Status Completed (Fiscal Year 2019)
Budget Amount *help
¥6,500,000 (Direct Cost: ¥5,000,000、Indirect Cost: ¥1,500,000)
Fiscal Year 2018: ¥2,730,000 (Direct Cost: ¥2,100,000、Indirect Cost: ¥630,000)
Fiscal Year 2017: ¥3,770,000 (Direct Cost: ¥2,900,000、Indirect Cost: ¥870,000)
KeywordsiPS細胞 / 遺伝性難聴 / 疾患iPS研究 / 創薬
Outline of Final Research Achievements

In the research, we focused on the hearing impairment of hair cells, which are responsible for the conversion of sound stimulation to nerve impulses, which is an essential function of the cochlea. We succeeded in establishing iPS cells from these 3 cases of hereditary deafness, and improved the differentiation induction protocol and established the electrophysiological method. These newly established reliable and robust protocol was being applied for the patent.

Academic Significance and Societal Importance of the Research Achievements

老人性難聴や突発性難聴、メニエール病など、感音難聴の多くは蝸牛障害による内耳性難聴で、本邦の身体障害者認定の約10%が難聴に関連している。本研究が開始された頃、次世代シークエンサーを用いた難聴遺伝子検査が保険収載され、過去に報告のない遺伝子変異(バリアント)が多数報告されるようになっていた。そのため、検査としての遺伝子解析は急速にハイスループット化しているにもかかわらず、得られた遺伝子変異の解釈を行う生物学的解析の進歩が追いついていない状態だった。本研究成果はこの問題を解決するソリューションとなり得るものであり、内耳性難聴の病態の把握や理解とともに、診断技術の向上に役立つ意義がある。

Report

(4 results)
  • 2019 Annual Research Report   Final Research Report ( PDF )
  • 2018 Research-status Report
  • 2017 Research-status Report
  • Research Products

    (45 results)

All 2020 2019 2018 2017 Other

All Journal Article (6 results) (of which Peer Reviewed: 5 results,  Open Access: 3 results) Presentation (35 results) (of which Int'l Joint Research: 11 results,  Invited: 9 results) Book (1 results) Remarks (3 results)

  • [Journal Article] 幹細胞生物学と霊長類モデルを用いた内耳性難聴の治療法開発2020

    • Author(s)
      藤岡 正人
    • Journal Title

      Otology Japan

      Volume: 30巻1号 Pages: 11-18

    • NAID

      130007890724

    • Related Report
      2019 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Comprehensive analysis of syndromic hearing loss patients in Japan2019

    • Author(s)
      Ideura Michie、Nishio Shin-ya、Sugahara Kazuma、他
    • Journal Title

      Scientific Reports

      Volume: 9 Issue: 1 Pages: 11976-11976

    • DOI

      10.1038/s41598-019-47141-4

    • Related Report
      2019 Annual Research Report
    • Peer Reviewed / Open Access
  • [Journal Article] 幹細胞生物学を応用した内耳性難聴治療法の開発研究2019

    • Author(s)
      藤岡 正人
    • Journal Title

      Otology Japan

      Volume: 29巻2号 Pages: 125-130

    • NAID

      130007751179

    • Related Report
      2019 Annual Research Report
    • Peer Reviewed
  • [Journal Article] Estimating the concentration of therapeutic range using disease-specific iPS cells: Low-dose rapamycin therapy for Pendred syndrome2018

    • Author(s)
      Hosoya M, Saeki T, Saegusa C, Matsunaga T, Okano H, Fujioka M, Ogawa K.
    • Journal Title

      Regen Ther

      Volume: 17;10 Pages: 54-63

    • DOI

      10.1016/j.reth.2018.11.001

    • Related Report
      2018 Research-status Report
    • Peer Reviewed / Open Access
  • [Journal Article] 【脳タンパク質老化と認知症制御】 iPSC及びマーモセットモデルを用いたPendred症候群のヒト特異的病態の解明と治療法開発2018

    • Author(s)
      佐伯翼,藤岡正人,岡野栄之
    • Journal Title

      細胞

      Volume: 50巻6号 Pages: 315-318

    • Related Report
      2018 Research-status Report
  • [Journal Article] Prevalence of TECTA mutation in patients with mid-frequency sensorineural hearing loss.2017

    • Author(s)
      Yamamoto N, Mutai H, Namba K, Morita N, Masuda S, Nishi Y, Nakano A, Masuda S, Fujioka M, Kaga K, Ogawa K, Matsunaga T.
    • Journal Title

      Orphanet J Rare Dis

      Volume: 25;12(1) Issue: 1 Pages: 157-157

    • DOI

      10.1186/s13023-017-0708-z

    • Related Report
      2017 Research-status Report
    • Peer Reviewed / Open Access
  • [Presentation] Selective and super-selective induction of cochlear hair cells from human iPS cells2020

    • Author(s)
      Tsubasa Saeki、Makoto Hosoya,Masato Fujioka,Kaoru Ogawa,Hideyuki Okano
    • Organizer
      ARO Abstract Confirmation 43rd MidWinter Meeting
    • Related Report
      2019 Annual Research Report
    • Int'l Joint Research
  • [Presentation] 当院における難聴遺伝外来の現況とこころみ2019

    • Author(s)
      細谷誠
    • Organizer
      第120回日本耳鼻咽喉科学会総会
    • Related Report
      2019 Annual Research Report
  • [Presentation] 幹細胞生物学と霊長類動物モデルを応用した内耳性難聴治療法の開発研究2019

    • Author(s)
      藤岡 正人
    • Organizer
      金沢医師会
    • Related Report
      2019 Annual Research Report
    • Invited
  • [Presentation] ヒトiPS細胞由来蝸牛オルガノイドの構築2019

    • Author(s)
      藤岡 正人
    • Organizer
      第40回炎症再生医学会
    • Related Report
      2019 Annual Research Report
  • [Presentation] Differentiation of human induced pluripotent stem cells into cochlear hair cells2019

    • Author(s)
      Tsubasa Saeki、Makoto Hosoya, Masato Fujioka, Kaoru Ogawa, Hideyuki Okano
    • Organizer
      NEURO2019
    • Related Report
      2019 Annual Research Report
  • [Presentation] ”新しい治療”を創り出す:産学官の"現場"の若手たち2019

    • Author(s)
      藤岡正人、武内 俊樹・黑川 友哉 ・阿部 圭悟・長倉 晃
    • Organizer
      第29回日本耳科学会総会・学術講演会
    • Related Report
      2019 Annual Research Report
    • Invited
  • [Presentation] Regeneration of auditory hair cells as a therapeutic for sensorineural hearing loss2019

    • Author(s)
      Masato Fujioka
    • Organizer
      第29回耳科学会総会・学術講演会
    • Related Report
      2019 Annual Research Report
    • Invited
  • [Presentation] ネクストジェネレーションセッション4: 幹細胞医学・再生医療の実用化研究:創薬の立場から2019

    • Author(s)
      藤岡 正人
    • Organizer
      第29回耳科学会総会・学術講演会
    • Related Report
      2019 Annual Research Report
    • Invited
  • [Presentation] 幹細胞生物学と霊長類モデルを用いた内耳性難聴の治療法開発 第2回日本耳科学会賞受賞者講演2019

    • Author(s)
      藤岡 正人
    • Organizer
      第29回耳科学会総会・学術講演会
    • Related Report
      2019 Annual Research Report
  • [Presentation] 化合物を用いたヒトiPS 細胞から内耳蝸牛感覚上皮への分化誘導条件の検討2019

    • Author(s)
      佐伯翼、細谷誠,藤岡正人,三枝智香,小川郁
    • Organizer
      第29回耳科学会総会・学術講演会
    • Related Report
      2019 Annual Research Report
  • [Presentation] Selective induction of cochlear hair cells from human induced pluripotent stem cells2019

    • Author(s)
      Tsubasa Saeki、Makoto Hosoya, Masato Fujioka, Kaoru Ogawa, Hideyuki Okano
    • Organizer
      56th. Inner Ear Biology Workshop
    • Related Report
      2019 Annual Research Report
    • Int'l Joint Research
  • [Presentation] 慢性感音難聴への挑戦 : iPS 創薬 シンポジウム2019

    • Author(s)
      藤岡正人
    • Organizer
      第64回日本聴覚医学会総会・学術講演会
    • Related Report
      2019 Annual Research Report
  • [Presentation] 幹細胞医学を応用した内耳再生医療創薬2019

    • Author(s)
      藤岡正人
    • Organizer
      神戸再生医療勉強会
    • Related Report
      2018 Research-status Report
    • Invited
  • [Presentation] 疾患 iPS 細胞でのプロテオミクスが明らかにした Pendred 症候群の新規病態メカニズム2019

    • Author(s)
      佐伯翼、細谷誠、芝田晋介、小川郁、藤岡正人、岡野栄之
    • Organizer
      第18回日本再生医療学会
    • Related Report
      2018 Research-status Report
  • [Presentation] Cochlear cell modeling using iPSCs unveils a degenerative phenotype and suggests traetments for hereditary hearing loss2018

    • Author(s)
      Hosoya M, Fujioka M, Sone T, Okamoto S, Akamatsu W, Ukai H, Ueda HR, Ogawa K, Matsunaga T, Okano H.
    • Organizer
      European Academy of Otology and Neurotology 2018
    • Related Report
      2018 Research-status Report
    • Int'l Joint Research
  • [Presentation] Highly Efficient, Well-reproducible Cochlear Organoids Derived from Human Induced Pluripotent Cells: A Candidate Method of the Drug Development for Spiral Ganglion Neuron2018

    • Author(s)
      Masato Fujioka, Tomohiko Yoshida, Makoto Hosoya, Kaoru Ogawa, Hiromi Kojima
    • Organizer
      European Academy of Otology and Neurotology 2018
    • Related Report
      2018 Research-status Report
    • Int'l Joint Research
  • [Presentation] 藤岡正人2018

    • Author(s)
      疾患iPS細胞を用いた難聴の創薬研究 動物モデル不在の病気へのチャレンジ
    • Organizer
      第39回日本炎症・再生医学会
    • Related Report
      2018 Research-status Report
  • [Presentation] Low-dose rapamycin therapy proposed by an in vitro chronic disorder model of Pendred Syndrome using disease specific iPS cells2018

    • Author(s)
      Masato Fujioka・Tsubasa Saeki・Kaoru Ogawa
    • Organizer
      55th Inner Ear Biology Workshop
    • Related Report
      2018 Research-status Report
    • Int'l Joint Research
  • [Presentation] Bridging rodent experiments to human clinical trials in gene therapy: non-human primate models and a novel DDS for rAAV mediated- gene transfe2018

    • Author(s)
      Masato Fujioka
    • Organizer
      International Symposium for Gene Tehrapy and Inherited Diseases
    • Related Report
      2018 Research-status Report
    • Int'l Joint Research
  • [Presentation] 幹細胞生物学を応用した内耳性難聴治療法の開発研究2018

    • Author(s)
      藤岡正人
    • Organizer
      第28回日本耳科学会総会・学術講演会
    • Related Report
      2018 Research-status Report
  • [Presentation] ヒトiPS細胞の内耳病態研究への応用と未来への展望~その長所・短所と位置付け~2018

    • Author(s)
      細谷誠、藤岡正人、小川郁
    • Organizer
      第28回日本耳科学会総会・学術講演会
    • Related Report
      2018 Research-status Report
  • [Presentation] In vitro modeling of hereditary hearing loss with human iPSC technology: Cellular pathology and drug discovery2018

    • Author(s)
      Hosoya M, Fujioka M, Matsunaga T, Ogawa K
    • Organizer
      AAO-HNSF Annual Meeting & OTO Experience
    • Related Report
      2018 Research-status Report
    • Int'l Joint Research
  • [Presentation] 遺伝性難聴Pendred症候群/DFNB4に対する疾患iPS創薬研究2018

    • Author(s)
      藤岡正人、細谷誠、佐伯翼、三枝智香、廣井有香、岡野栄之、小川郁
    • Organizer
      第63回日本人類遺伝学大会
    • Related Report
      2018 Research-status Report
  • [Presentation] 幹細胞生物学を応用した内耳性難聴の研究2018

    • Author(s)
      藤岡正人
    • Organizer
      ディレグラWebセミナー
    • Related Report
      2018 Research-status Report
    • Invited
  • [Presentation] 小型霊長類コモンマーモセット内耳発生の解析: 霊長類有毛細胞への再生医学的アプローチの基礎的知見として2018

    • Author(s)
      細谷 誠,藤岡正人,小川 郁
    • Organizer
      第63回日本聴覚医学会・学術講演会
    • Related Report
      2018 Research-status Report
  • [Presentation] iPS創薬の実際: 感音難聴の治療法開発2018

    • Author(s)
      藤岡正人
    • Organizer
      第17回ENT病診連携カンファレンス
    • Related Report
      2018 Research-status Report
    • Invited
  • [Presentation] 両側進行性難聴の病態解明と新規治療法開発~iPS創薬の立場から~2017

    • Author(s)
      藤岡正人
    • Organizer
      第17回日本抗加齢医学会総会
    • Related Report
      2017 Research-status Report
  • [Presentation] Cochlear cell modeling using disease-specific iPSCs unveiled a degenerative phenotype and treatments of a congenital progressive hearing loss2017

    • Author(s)
      Makoto Hosoya,Masato Fujioka , Tatsuo Matsunaga , Kaoru Ogawa ,
    • Organizer
      ENT World Congress IFOS Paris 2017
    • Related Report
      2017 Research-status Report
    • Int'l Joint Research
  • [Presentation] 『両側進行性難聴に対するiPS細胞研究~モデル動物不在の疾患への挑戦~』2017

    • Author(s)
      藤岡正人
    • Organizer
      第16回バイオ・ライフサイエンス研究展BIOtech
    • Related Report
      2017 Research-status Report
  • [Presentation] 内耳性難聴に対するiPS細胞を用いた創薬研究2017

    • Author(s)
      藤岡正人
    • Organizer
      第38回大阪耳疾患セミナー
    • Related Report
      2017 Research-status Report
  • [Presentation] Cochlear Cell Modeling Using Disease-Specific iPSCs Unveils a Degenerative Phenotype and Suggests Treatments for Congenital Progressive Hearing Loss2017

    • Author(s)
      Masato Fujioka
    • Organizer
      8 th World Gene Convention-2017
    • Related Report
      2017 Research-status Report
    • Int'l Joint Research
  • [Presentation] Pendred症候群患者特異的iPS細胞由来内耳細胞におけるPENDRINタンパク質の局在の検討2017

    • Author(s)
      佐伯翼・藤岡正人・細谷誠・西山崇経・松崎佐栄子・松永達雄・小川郁
    • Organizer
      第27回日本耳科学会総会・学術講演会
    • Related Report
      2017 Research-status Report
  • [Presentation] EYA4遺伝子変異特異的疾患iPS細胞の樹立2017

    • Author(s)
      松崎佐栄子・藤岡正人・細谷誠・佐伯翼・阿部聡子・松永達雄・宇佐美真一・小川郁
    • Organizer
      第27回日本耳科学会総会・学術講演会
    • Related Report
      2017 Research-status Report
  • [Presentation] Predicting clinical effects of therapeutics by iPS-based in vitro model of genetic deafness: another promising approach of precision medicine2017

    • Author(s)
      Masato Fujioka
    • Organizer
      国際検討会 Presicion Medicine For Deafness
    • Related Report
      2017 Research-status Report
    • Int'l Joint Research / Invited
  • [Presentation] Therapeutics in genetic deafness by iPS-based in vitro model2017

    • Author(s)
      Masato Fujioka
    • Organizer
      臺大醫院演講
    • Related Report
      2017 Research-status Report
    • Int'l Joint Research / Invited
  • [Book] 【難聴をなおす-2020年版】感音難聴とiPS創薬2020

    • Author(s)
      藤岡正人、細谷誠
    • Total Pages
      128
    • Publisher
      JOHNS
    • ISBN
      9784885635274
    • Related Report
      2019 Annual Research Report
  • [Remarks] 慶應義塾大学医学部耳鼻咽喉科学教室研究紹介

    • Related Report
      2019 Annual Research Report
  • [Remarks] 慶應義塾大学医学部耳鼻咽喉科学教室研究紹介

    • Related Report
      2018 Research-status Report
  • [Remarks] 慶應義塾大学医学部耳鼻咽喉科学教室研究紹介

    • Related Report
      2017 Research-status Report

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Published: 2017-07-21   Modified: 2022-12-28  

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