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2022 Fiscal Year Final Research Report

Overhanging duplex oligonucleotide penetrating BBB

Research Project

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Project/Area Number 20K21882
Research Category

Grant-in-Aid for Challenging Research (Exploratory)

Allocation TypeMulti-year Fund
Review Section Medium-sized Section 90:Biomedical engineering and related fields
Research InstitutionTokyo Medical and Dental University

Principal Investigator

YOSHIOKA KOTARO  東京医科歯科大学, 大学院医歯学総合研究科, 特任助教 (70780641)

Project Period (FY) 2020-07-30 – 2023-03-31
Keywordsアンチセンス核酸 / 脳血液関門
Outline of Final Research Achievements

Therapeutic oligonucleotides, as the next generation of medicine following antibody therapeutics, has rapidly developed in clinical research. However, targeted organ delivery of the nucleotides has depended on the characteristics of delivery vehicles, and their toxicity remains a significant concern. The researcher has developed a novel type of double-stranded nucleic acid named overhang Double-stranded Oligonucleotide (ODO), which incorporates a protruding complementary nucleic acid strand into conventional single-stranded antisense nucleotides. The protruding nucleic acid molecule itself serves as a delivery carrier. By optimizing the chain length and nucleic acid modifications in the overhang structure, as well as discovering interchain modifications and sugar chemistry modifications that greatly enhance transferrin binding ability, researchers have successfully improved the in vivo gene inhibition effect.

Free Research Field

核酸医薬

Academic Significance and Societal Importance of the Research Achievements

従来の核酸医薬とは全く分子構造が異なるODOはリガンド分子を用いないトランスフェリン介在性細胞内取り込み能という革新性を有しており、本研究により中枢神経標的の核酸医薬のブレイクスルーを起こし、アルツハイマー病などの神経難病やうつ病など超高齢社会を迎えて健康寿命を脅かす神経精神疾患の根本治療開発への大きな波及効果が期待される

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Published: 2024-01-30  

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