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2023 Fiscal Year Final Research Report

Investigation into neuronal repair and functional recovery in neurodegenerative diseases

Research Project

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Project/Area Number 21H02819
Research Category

Grant-in-Aid for Scientific Research (B)

Allocation TypeSingle-year Grants
Section一般
Review Section Basic Section 51030:Pathophysiologic neuroscience-related
Research InstitutionTokyo Metropolitan Institute of Medical Science

Principal Investigator

HARADA Takayuki  公益財団法人東京都医学総合研究所, 疾患制御研究分野, 参事研究員 (90345306)

Co-Investigator(Kenkyū-buntansha) 行方 和彦  公益財団法人東京都医学総合研究所, 疾患制御研究分野, 副参事研究員 (70392355)
原田 知加子  公益財団法人東京都医学総合研究所, 疾患制御研究分野, 研究員 (20435720)
Project Period (FY) 2021-04-01 – 2024-03-31
Keywords神経変性疾患 / 遺伝子治療 / 軸索再生 / 視神経再生
Outline of Final Research Achievements

Neurodegenerative diseases progress due to synapse loss and cell death caused by neurite retraction. Therefore, an adeno-associated viral vector (AAV-iTrkB) was created to force expression of the intracellular region of TrkB, a high-affinity receptor for brain-derived neurotrophic factor (BDNF), at the plasma membrane. When AAV-iTrkB was administered to an optic nerve trauma model, regeneration of optic nerve axons was observed. When administered to a glaucoma model, cell death was suppressed. Furthermore, in an optic tract transection model, regenerating axons successfully formed pathways to their brain targets, resulting in partial recovery of visual behavior. These results indicate that gene therapy using AAV-iTrkB vector may contribute to neurite outgrowth and recovery of neuronal function.

Free Research Field

神経科学

Academic Significance and Societal Importance of the Research Achievements

視神経外傷や緑内障などで視神経が変性した場合、現在のところ直接的な治療法は開発されていない。今回作製した遺伝子治療ベクター (AAV-iTrkB)はBDNFの投与を必要としない画期的なものであり、視神経の保護や再生にも強力な効果を示した。この成果は国際的な専門誌において発表済み(Molecular Therapy, 2023)であり、国際特許申請も終えている。またこうした新しい手法は他の神経変性疾患の治療にも応用可能であることから、将来的に大きな社会的意義を持つことが期待される。

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Published: 2025-01-30  

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