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Review
. 2017 Oct;31(5):787-795.
doi: 10.1016/j.hoc.2017.05.002. Epub 2017 Jun 29.

Therapeutic Gene Editing Safety and Specificity

Christopher T Lux  1 , Andrew M Scharenberg  2
Affiliations
Review

Therapeutic Gene Editing Safety and Specificity

Christopher T Lux et al. Hematol Oncol Clin North Am. 2017 Oct.

Abstract

Therapeutic gene editing is significant for medical advancement. Safety is intricately linked to the specificity of the editing tools used to cut at precise genomic targets. Improvements can be achieved by thoughtful design of nucleases and repair templates, analysis of off-target editing, and careful utilization of viral vectors. Advancements in DNA repair mechanisms and development of new generations of tools improve targeting of specific sequences while minimizing risks. It is important to plot a safe course for future clinical trials. This article reviews safety and specificity for therapeutic gene editing to spur dialogue and advancement.

Keywords: Gene editing; Gene therapy; Off-target; Safety; Specific.

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