Recent advances in the use of ZFN-mediated gene editing for human gene therapy

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Expert Insight

Srinivasan Chandrasegaran

Gene Editing Technologies

Targeted genome editing with programmable nucleases has revolutionized biomedical research. The ability to make site-specific modifications to the human genome, has invoked a paradigm shift in gene therapy. Using gene editing technologies, the sequence in the human genome can now be precisely engineered to achieve a therapeutic effect. Zinc finger nucleases (ZFNs) were the first programmable nucleases designed to target and cleave custom sites. This article summarizes the advances in the use of ZFN-mediated gene editing for human gene therapy and discusses the challenges associated with translating this gene editing technology into clinical use.

Published: Feb 8 2017
DOI: 10.18609/cgti.2017.005
Citation: Cell Gene Therapy Insights 2017;3(1), 33-41.
Open access

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