RNAの世界からクリニックへ
Bruce A Sullenger1, Smita Nair1
1Duke Translational Research Institute and Department of Surgery, Duke University Medical Center, Durham, NC 27710, USA.
Science (New York, N.Y.)
|June 18, 2016
まとめ
RNA治療は様々な病気の治療に 有望な結果を示しています 臨床試験では,癌のためのメッセンジャーRNA,腎臓と肝臓の問題のための小さな干渉RNA,および眼と心臓の疾患のためのアプタマーを調査しています.
科学分野:
- 生物化学
- 分子生物学
- 治療薬
背景:
- RNA分子は 驚くべき構造的・機能的な 多様性を持っています
- この汎用性は,様々な医療用途のためのRNAベースの治療薬の研究を推進しています.
研究 の 目的:
- 臨床実務におけるRNA治療の役割と可能性を強調する.
- RNAベースの薬の開発の現状をまとめます
主な方法:
- 様々なRNAの治療方法に関する進行中の臨床試験のレビュー.
- 患者登録と治療目標の分析 末期と初期段階の発達
主要な成果:
- メッセンジャーRNA,小干渉RNA,アプタマーを含む複数のRNA治療薬は,臨床試験の最終段階にある.
- 何百人もの患者ががん,腎臓,肝臓疾患,眼,心血管疾患の治験に参加しています.
結論:
- RNA治療薬は,重要な臨床的可能性を持つ迅速に進歩する薬剤のクラスを表しています.
- 治験の陽性な結果は,RNAベースの薬の将来的な開発と採用を推進すると予想されます.
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