Title:CRISPR/Cas9 Gene Editing: From Basic Mechanisms to Improved Strategies for Enhanced Genome Engineering In Vivo
VOLUME: 17 ISSUE: 4
Author(s):Jayme Salsman, Jean-Yves Masson, Alexandre Orthwein* and Graham Dellaire*
Affiliation:Department of Pathology, Dalhousie University, Halifax, NS, Genome Stability Laboratory, CHU de Quebec Research Center, Quebec City, QC, Lady Davis Institute for Medical Research, Segal Cancer Centre, Jewish General Hospital, Montreal, QC, Department of Pathology, Dalhousie University, Halifax, NS
Keywords:CRISPR-Cas9, Gene editing, Non-homologous end-joining, Homologous recombination, DNA repair.
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.
