核酸医薬の基礎と臨床　総論・化学

DOI
  • 原 倫太朗
    東京医科歯科大学大学院医歯学総合研究科脳神経病態学分野
  • 横田 隆徳
    東京医科歯科大学大学院医歯学総合研究科脳神経病態学分野

書誌事項

タイトル別名
  • Chemistry and clinical application in oligonucleotide therapeutics

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説明

<p>Oligonucleotide therapeutics are based on chemically synthesized oligonucleotides which are in almost all cases chemically modified. They are classified into some categories by their action mechanisms, such as antisense oligonucleotides (ASOs) and RNA interference (RNAi) drugs. These oligonucleotides can bind to their target molecules such as mRNAs by Watson–Click base pairing and/or other interaction also including multipoint hydrogen bonding. By their definitive action mechanisms and their molecular size which are between small molecules and antibody drug, oligonucleotide therapeutics can be highly–specific drugs that can target molecules in cells. In this review, we outline the classification and action mechanisms of oligonucleotide therapeutics. In addition, we also introduce nucleic acid chemistry, which can make biologically–unstable nucleic acid molecules into effective therapeutic reagents.</p>

収録刊行物

  • 神経治療学

    神経治療学 37 (3), 238-241, 2020

    日本神経治療学会

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