CRISPR/Cas9 gene editing: From basic mechanisms to improved strategies for enhanced genome engineering in vivo

Jayme Salsman; Jean Yves Masson; Alexandre Orthwein; Graham Dellaire

doi:10.2174/1566523217666171122094629

CRISPR/Cas9 gene editing: From basic mechanisms to improved strategies for enhanced genome engineering in vivo

Jayme Salsman, Jean Yves Masson, Alexandre Orthwein, Graham Dellaire

Medicine

Producción científica: Contribución a una revista › Artículo de revisión › revisión exhaustiva

15 Citas (Scopus)

Resumen

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.

Idioma original	English
Páginas (desde-hasta)	263-274
Número de páginas	12
Publicación	Current Gene Therapy
Volumen	17
N.º	4
DOI	https://doi.org/10.2174/1566523217666171122094629
Estado	Published - 2017

Nota bibliográfica

Funding Information:
This work was supported in part by research grants from the Canadian Institutes of Health Research (CIHR) to GD (MOP-84260), AO (PJ/361708 and PJ/376245), and JYM (PJ/375408), the Natural Sciences and Engineering Research Council of Canada (NSERC) to GD (RGPIN/05616), the Cole Foundation and the Sir Mortimer B. Davis Foundation from the Jewish General Hospital for AO. GD is a Senior Scientist of the Beatrice Hunter Cancer Research Institute (BHCRI) and this work was also supported in part by a Bridge grant from BHCRI to GD. J.S. was a recipient of a BHCRI postdoctoral fellowship made possible by the Harvey Graham Cancer Research Fund as part of the Terry Fox Foundation Strategic Health Research Training Program in Cancer Research at CIHR. AO is a Canada Research Chair Tier 2 in Genome Stability and Haematological malignancies.

Publisher Copyright:
© 2017 Bentham Science Publishers.

ASJC Scopus Subject Areas

Molecular Medicine
Molecular Biology
Genetics
Drug Discovery
Genetics(clinical)

PubMed: MeSH publication types

Journal Article
Research Support, Non-U.S. Gov't
Review

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abstract = "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.",

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CRISPR/Cas9 gene editing: From basic mechanisms to improved strategies for enhanced genome engineering in vivo

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