Cardiff University | Prifysgol Caerdydd ORCA
Online Research @ Cardiff 
WelshClear Cookie - decide language by browser settings

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

Mills, Emily M., Barlow, Victoria L., Luk, Louis Y. P. ORCID: https://orcid.org/0000-0002-7864-6261 and Tsai, Yu-Hsuan ORCID: https://orcid.org/0000-0003-0589-5088 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

[thumbnail of Mills2020_Article_ApplyingSwitchableCas9Variants.pdf]
Preview
PDF - Published Version
Available under License Creative Commons Attribution.

Download (997kB) | Preview
License URL: http://creativecommons.org/licenses/by/4.0
License Start date: 15 August 2019

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: 07 Jul 2023 18:19
URI: https://orca.cardiff.ac.uk/id/eprint/125063

Citation Data

Cited 6 times in Scopus. View in Scopus. Powered By Scopus® Data

Actions (repository staff only)

Edit Item Edit Item

Downloads

Downloads per month over past year

View more statistics