核酸医薬の基礎と臨床 総論
書誌事項
- タイトル別名
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- Basic principles and clinical applications in oligonucleotide therapeutics
説明
<p>Oligonucleotide therapeutics belong to molecularly–targeted therapy as well as antibody drugs and gene therapy drugs do, and directly bind target molecule without gene expression. They can bind nuclear RNA and work in the cell, though the antibody drug targets are limited to cell surface molecules. Antisense oligonucleotides, small interfering RNA, decoy, aptamer, and CpG ologonucleotides are mojor subtypes of oligonucleotide therapeutics which are classifed by the mechnism of action. The improvement of chemical modifications such as backbone modificatins and sugar modifications have increased thier stability. As a result, many of FDA approved oligonucleotide therapeutics have been developed in past five years. In 2020, viltolarsen was approved by FDA as a first antisense oligonucleotide developed in Japan. In this abstract, we review the characteristics, classification, and chemical modifications of oligonucleotide therapeutics. Moreover, we outline their current status in clinical practice.</p>
収録刊行物
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- 神経治療学
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神経治療学 38 (3), 257-260, 2021
日本神経治療学会
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キーワード
詳細情報 詳細情報について
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- CRID
- 1390009305281274624
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- NII論文ID
- 130008142609
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- ISSN
- 21897824
- 09168443
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- 本文言語コード
- ja
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- データソース種別
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- JaLC
- CiNii Articles
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- 抄録ライセンスフラグ
- 使用不可
