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

ABSTRACT: 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.

SUBMITTER: Chandrasegaran S

PROVIDER: S-EPMC5736148 | biostudies-literature | 2017

REPOSITORIES: biostudies-literature

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

Chandrasegaran Srinivasan S

Cell & gene therapy insights 20170108 1

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 ...[more]

PMID: 29270315

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