title: Application of CRISPR/Cas9 genome editing to the study and treatment of disease.
abstract: CRISPR/Cas is a microbial adaptive immune system that uses RNA-guided nucleases to cleave foreign genetic elements. The CRISPR/Cas9 method has been engineered from the type II prokaryotic CRISPR system and uses a single-guide RNA to target the Cas9 nuclease to a specific genomic sequence. Cas9 induces double-stranded DNA breaks which are repaired either by imperfect non-homologous end joining to generate insertions or deletions (indels) or, if a repair template is provided, by homology-directed repair. Due to its specificity, simplicity and versatility, the CRISPR/Cas9 system has recently emerged as a powerful tool for genome engineering in various species. This technology can be used to investigate the function of a gene of interest or to correct gene mutations in cells via genome editing, paving the way for future gene therapy approaches. Improvements to the efficiency of CRISPR repair, in particular to increase the rate of gene correction and to reduce undesired off-target effects, and the development of more effective delivery methods will be required for its broad therapeutic application.
authors.affiliation: Leukaemia and Lymphoma Research Molecular Haematology Unit, Nuffield Division of Clinical Laboratory Sciences, Radcliffe Department of Medicine, and NIHR Biomedical Research Centre, Oxford University Hospitals, University of Oxford, Oxford, OX3 9DU, UK.
authors.forename: Andrea
authors.initials: A
authors.lastname: Pellagatti
authors.forename: Hamid
authors.initials: H
authors.lastname: Dolatshad
authors.forename: Simona
authors.initials: S
authors.lastname: Valletta
authors.forename: Jacqueline
authors.initials: J
authors.lastname: Boultwood
mesh_terms: D000818:Animals; D064130:CRISPR-Associated Proteins / Q000235:genetics* / Q000378:metabolism; D064113:CRISPR-Cas Systems; D064112:Clustered Regularly Interspaced Short Palindromic Repeats; D005786:Gene Expression Regulation; D018014:Gene Transfer Techniques; D005818:Genetic Engineering / Q000379:methods*; D015316:Genetic Therapy / Q000379:methods*; D006801:Humans; D017393:RNA Editing
chemical_list: D064130:CRISPR-Associated Proteins