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Therapeutic Gene Editing Safety and Specificity. | LitMetric

Therapeutic Gene Editing Safety and Specificity.

Hematol Oncol Clin North Am

Department of Pediatrics, Seattle Children's Hospital, 4800 Sand Point Way NE, Seattle, WA 98105, USA; Department of Immunology, Seattle Children's Hospital, 4800 Sand Point Way NE, Seattle, WA 98105, USA. Electronic address:

Published: October 2017

AI Article Synopsis

Article 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.

Download full-text PDF

Source
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC5653273PMC
http://dx.doi.org/10.1016/j.hoc.2017.05.002DOI Listing

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