CRISPR / Cas9基因编辑：从基本机制到改进的基因组工程体内策略

Title:CRISPR/Cas9 Gene Editing: From Basic Mechanisms to Improved Strategies for Enhanced Genome Engineering In Vivo

Volume: 17 Issue: 4

关键词: CRISPR-Cas9，基因编辑，非同源末端连接，同源重组，DNA修复。

摘要: Introduction: Targeted genome editing using the CRISPR/Cas9 technology is becoming a major area of research due to its high potential for the treatment of genetic diseases. Our understanding of this approach has expanded in recent years yet several new challenges have presented themselves as we explore the boundaries of this exciting new technology. Chief among these is improving the efficiency but also the preciseness of genome editing. The efficacy of CRISPR/Cas9 technology relies in part on the use of one of the major DNA repair pathways, Homologous recombination (HR), which is primarily active in S and G2 phases of the cell cycle. Problematically, the HR potential is highly variable from cell type to cell type and most of the cells of interest to be targeted in vivo for precise genome editing are in a quiescent state.

Conclusion: In this review, we discuss the recent advancements in improving targeted CRISPR/Cas9 based genome editing and the promising ways of delivering this technology in vivo to the cells of interest.

Cite this article as:

CRISPR/Cas9 Gene Editing: From Basic Mechanisms to Improved Strategies for Enhanced Genome Engineering In Vivo, Current Gene Therapy 2017; 17 (4) . https://dx.doi.org/10.2174/1566523217666171122094629