ゲノム工学のCRISPR-Cas9の開発と応用について

Patrick D Hsu¹, Eric S Lander², Feng Zhang³

¹Broad Institute of MIT and Harvard, 7 Cambridge Center, Cambridge, MA 02141, USA; McGovern Institute for Brain Research, Department of Brain and Cognitive Sciences, Department of Biological Engineering, Massachusetts Institute of Technology, Cambridge, MA 02139, USA; Department of Molecular and Cellular Biology, Harvard University, Cambridge, MA 02138, USA.

Cell

|June 7, 2014

まとめ

CRISPR-Cas9遺伝子編集技術は,科学者がDNA配列を正確に修正することを可能にします. この強力なツールは,研究者がゲノム機能を研究し,遺伝子の多様性を理解することを可能にします.

科学分野:

ゲノミクスゲノミクスとは
分子生物学は分子生物学である.
バイオテクノロジーバイオテクノロジー

背景:

CRISPR関連 (Cas) システム,特にCas9は,微生物の防御メカニズムである.
これらのシステムは,標的型DNA操作のためにRNA誘導型エンドヌクレアスを利用します.
最近の進歩により,哺乳類のゲノム機能の体系的な尋問が可能になりました.

研究の目的:

Cas9技術の開発と応用をレビューする.
ゲノム工学と機能的ゲノミクスにおけるCas9の役割を強調する.
Cas9ベースの研究における課題と将来の方向性について議論する.

主な方法:

Cas9はRNAによって特定のゲノム部位に誘導される.
固有のゲノムにおけるDNA配列の編集または調節.
システムレベルの分析のためのスケーラブルな遺伝的混乱.

主要な成果:

Cas9は,様々な生物のDNAを正確に編集することを可能にします.
ゲノム組織と機能の解明を容易にする.
遺伝的変異とフェノタイプとの間の因果関係を確立する.

結論:

Cas9技術は,基礎生物学,バイオテクノロジー,医学に革命を起こしています.
そのシンプルさとスケーラビリティは,包括的なゲノム研究を可能にします.
進行中の研究により,さらなる革新的な応用が期待されています.

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