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

Author(s): Jayme Salsman, Jean-Yves Masson, Alexandre Orthwein*, Graham Dellaire*

Journal Name: Current Gene Therapy

Volume 17 , Issue 4 , 2017

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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.

Keywords: CRISPR-Cas9, Gene editing, Non-homologous end-joining, Homologous recombination, DNA repair.

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Article Details

Year: 2017
Published on: 04 January, 2018
Page: [263 - 274]
Pages: 12
DOI: 10.2174/1566523217666171122094629
Price: $65

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PDF: 38