Journal
BRIEFINGS IN FUNCTIONAL GENOMICS
Volume 16, Issue 1, Pages 4-12Publisher
OXFORD UNIV PRESS
DOI: 10.1093/bfgp/elw025
Keywords
genome engineering; gene editing; CRISPR; Cas9; human disease; cancer; animal models; biomedicine
Funding
- FIS project by Spanish National Research and Development Plan, Instituto de Salud Carlos III [PI14/01884]
- FEDER
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Genome engineering is a powerful tool for a wide range of applications in biomedical research and medicine. The development of the clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 system has revolutionized the field of gene editing, thus facilitating efficient genome editing through the creation of targeted double-strand breaks of almost any organismand cell type. In addition, CRISPR-Cas9 technology has been used successfully for many other purposes, including regulation of endogenous gene expression, epigenome editing, live-cell labelling of chromosomal loci, edition of singlestranded RNA and high-throughput gene screening. The implementation of the CRISPR-Cas9 system has increased the number of available technological alternatives for studying gene function, thus enabling generation of CRISPR-based disease models. Although many mechanistic questions remain to be answered and several challenges have yet to be addressed, the use of CRISPR-Cas9-based genome engineering technologies will increase our knowledge of disease processes and their treatment in the near future.
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