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研究分野生物医学と臨床科学腫瘍学とがん発生分子標的
人間の胚の中での Cas9: 目標に達しても修復できない

人間の胚の中での Cas9: 目標に達しても修復できない

Eva R Hoffmann ¹, Ignasi Roig ²

Eva R Hoffmann ¹, Ignasi Roig ²

¹Danish National Research Foundation Center for Chromosome Stability, Department of Cellular and Molecular Biology, Faculty of Health and Medical Sciences, University of Copenhagen, Denmark.
²Unitat de Citologia i Histologia, Departament de Biologia Cel·lular, Fisiologia i Immunologia, Facultat de Biociències, Universitat Autònoma de Barcelona, Cerdanyola del Vallès, Spain; Grup d'Inestabilitat i Integritat del Genoma, Institut de Biotecnologia i Biomedicina, Universitat Autònoma de Barcelona, Cerdanyola del Vallès, Spain.

⁰Danish National Research Foundation Center for Chromosome Stability, Department of Cellular and Molecular Biology, Faculty of Health and Medical Sciences, University of Copenhagen, Denmark.

Cell +

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2020年12月11日

関連する実験動画

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Using Next Generation Sequencing to Identify Mutations Associated with Repair of a CAS9-induced Double Strand Break Near the CD4 Promoter

Using Next Generation Sequencing to Identify Mutations Associated with Repair of a CAS9-induced Double Strand Break Near the CD4 Promoter

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A Standard Methodology to Examine On-site Mutagenicity As a Function of Point Mutation Repair Catalyzed by CRISPR/Cas9 and SsODN in Human Cells

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PubMedで要約を見る

まとめ

この要約は機械生成です。

CRISPR-Cas9遺伝子の編集は,研究されたヒト胚の90%以上で染色体の損失または誤った修復を引き起こしました. これは,支配的な末端結合修復経路による遺伝子手術の応用における重大なリスクを強調しています.

科学分野

分子生物学
遺伝学
遺伝子編集技術

背景

CRISPR-Cas9は強力な遺伝子編集ツールです
遺伝子編集は遺伝疾患を修正することを目的としています
Cas9による損傷の修復メカニズムを理解することは極めて重要です.

研究の目的

ヒト胚におけるCas9媒介による二重鎖断裂の結果を調査する.
Cas9の活性に伴う遺伝子修復経路の効率と精度を評価する.
ヒト胚における治療用遺伝子編集のための二重鎖断裂の使用の安全性と実現可能性を評価する.

主な方法

CRISPR-Cas9技術を活用して人間の胚に標的を絞った二重鎖の断絶を誘導した.
Cas9誘導後の染色体整合性とDNA修復の結果を分析した.
染色体の喪失,誤った修復,挿入/削除の頻度を定量化した.

主要な成果

標的のCas9媒介による二重鎖断裂は,ヒト胚の90%以上で染色体喪失または疾患アレルの誤修復をもたらした.
末端結合修復経路が優勢で,小さな挿入や削除につながった.
精密な遺伝子修正の効率が著しく低下した.

結論

CRISPR- Cas9によって誘発された二重鎖の断裂は,高確率の染色体異常と不正確な修復をヒト胚で引き起こします.
末端結合修復の優位性は,二重鎖の断裂に依存する治療用遺伝子編集戦略に重大な課題をもたらします.
現在,ヒト胚の"遺伝子手術"に用いられている二重鎖の断絶は,重大なリスクと限界を伴う.

関連する実験動画

自動的に一致したビデオです Contact us もしそれらが関連していない場合

Using Next Generation Sequencing to Identify Mutations Associated with Repair of a CAS9-induced Double Strand Break Near the CD4 Promoter

Using Next Generation Sequencing to Identify Mutations Associated with Repair of a CAS9-induced Double Strand Break Near the CD4 Promoter

Published on: March 31, 2022

A Standard Methodology to Examine On-site Mutagenicity As a Function of Point Mutation Repair Catalyzed by CRISPR/Cas9 and SsODN in Human Cells

A Standard Methodology to Examine On-site Mutagenicity As a Function of Point Mutation Repair Catalyzed by CRISPR/Cas9 and SsODN in Human Cells

Published on: August 25, 2017

Introducing Point Mutations into Human Pluripotent Stem Cells Using Seamless Genome Editing

Introducing Point Mutations into Human Pluripotent Stem Cells Using Seamless Genome Editing

Published on: May 10, 2020

See all related videos

関連する概念動画

CRISPR

CRISPR

Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...

In-vitro Mutagenesis

In-vitro Mutagenesis

To learn more about the function of a gene, researchers can observe what happens when the gene is inactivated or “knocked out,” by creating genetically engineered knockout animals. Knockout mice have been particularly useful as models for human diseases such as cancer, Parkinson’s disease, and diabetes.

The Process

Genes can be randomly knocked out, or specific genes can be targeted. To knock out a particular gene, an engineered piece of DNA called a targeting vector is used...

Homologous Recombination

Homologous Recombination

The basic reaction of homologous recombination (HR) involves two chromatids that contain DNA sequences sharing a significant stretch of identity. One of these sequences uses a strand from another as a template to synthesize DNA in an enzyme-catalyzed reaction. The final product is a novel amalgamation of the two substrates. To ensure an accurate recombination of sequences, HR is restricted to the S and G2 phases of the cell cycle. At these stages, the DNA has been replicated already and the...

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