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Applying switchable Cas9 variants to in vivo gene editing for therapeutic applications

Mills, Emily M., Barlow, Victoria L., Luk, Louis Y. P. and Tsai, Yu-Hsuan 2020. Applying switchable Cas9 variants to in vivo gene editing for therapeutic applications. Cell Biology and Toxicology 36 , pp. 17-29. 10.1007/s10565-019-09488-2

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Abstract

Progress in targeted gene editing by programmable endonucleases has paved the way for their use in gene therapy. Particularly, Cas9 is an endonuclease with high activity and flexibility, rendering it an attractive option for therapeutic applications in clinical settings. Many disease-causing mutations could potentially be corrected by this versatile new technology. In addition, recently developed switchable Cas9 variants, whose activity can be controlled by an external stimulus, provide an extra level of spatiotemporal control on gene editing and are particularly desirable for certain applications. Here, we discuss the considerations and difficulties for implementing Cas9 to in vivo gene therapy. We put particular emphasis on how switchable Cas9 variants may resolve some of these barriers and advance gene therapy in the clinical setting.

Item Type: Article
Date Type: Publication
Status: Published
Schools: Chemistry
Publisher: Springer Verlag (Germany)
ISSN: 0742-2091
Funders: Biotechnology and Biological Sciences Research Council, Wellcome Trust
Date of First Compliant Deposit: 22 August 2019
Date of Acceptance: 26 July 2019
Last Modified: 29 Apr 2020 14:39
URI: http://orca.cf.ac.uk/id/eprint/125063

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