Therapeutic Gene Editing Safety and Specificity

Overview

Journal Hematol Oncol Clin North Am

Specialties Hematology
Oncology

Date 2017 Sep 13

PMID 28895847

Citations 14

Authors

Christopher T Lux

Andrew M Scharenberg

Affiliations

Soon will be listed here.

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.

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