Salient Features of Endonuclease Platforms for Therapeutic Genome Editing

Molecular Therapy : the Journal of the American Society of Gene Therapy
Michael T Certo, Richard A Morgan

Abstract

Emerging gene-editing technologies are nearing a revolutionary phase in genetic medicine: precisely modifying or repairing causal genetic defects. This may include any number of DNA sequence manipulations, such as knocking out a deleterious gene, introducing a particular mutation, or directly repairing a defective sequence by site-specific recombination. All of these edits can currently be achieved via programmable rare-cutting endonucleases to create targeted DNA breaks that can engage and exploit endogenous DNA repair pathways to impart site-specific genetic changes. Over the past decade, several distinct technologies for introducing site-specific DNA breaks have been developed, yet the different biological origins of these gene-editing technologies bring along inherent differences in parameters that impact clinical implementation. This review aims to provide an accessible overview of the various endonuclease-based gene-editing platforms, highlighting the strengths and weakness of each with respect to therapeutic applications.

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Oct 22, 2016·Journal of Equine Veterinary Science·James West, W Warren Gill
Sep 9, 2016·Molecular Therapy. Methods & Clinical Development·Arnold ParkBenhur Lee
Dec 22, 2017·Veterinary World·Shakil Ahmad BhatNadeem Shabir
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Jun 25, 2021·Nature Communications·Liyang ZhangChristopher A Vakulskas

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