title: Advances in therapeutic CRISPR/Cas9 genome editing.
abstract: Targeted nucleases are widely used as tools for genome editing. Two years ago the clustered regularly interspaced short palindromic repeat (CRISPR)-associated Cas9 nuclease was used for the first time, and since then has largely revolutionized the field. The tremendous success of the CRISPR/Cas9 genome editing tool is powered by the ease design principle of the guide RNA that targets Cas9 to the desired DNA locus, and by the high specificity and efficiency of CRISPR/Cas9-generated DNA breaks. Several studies recently used CRISPR/Cas9 to successfully modulate disease-causing alleles in vivo in animal models and ex vivo in somatic and induced pluripotent stem cells, raising hope for therapeutic genome editing in the clinics. In this review, we will summarize and discuss such preclinical CRISPR/Cas9 gene therapy reports.
authors.affiliation: Institute of Molecular Health Sciences, ETH Zurich, Zurich, Switzerland.
authors.forename: Nataša
authors.initials: N
authors.lastname: Savić
authors.affiliation: Institute of Molecular Health Sciences, ETH Zurich, Zurich, Switzerland. Electronic address: schwankg@ethz.ch.
authors.forename: Gerald
authors.initials: G
authors.lastname: Schwank
mesh_terms: D000818:Animals; D001426:Bacterial Proteins / Q000235:genetics*; D000076987:CRISPR-Associated Protein 9; D064113:CRISPR-Cas Systems / Q000235:genetics* / Q000502:physiology; D064112:Clustered Regularly Interspaced Short Palindromic Repeats / Q000235:genetics / Q000502:physiology*; D004720:Endonucleases / Q000235:genetics*; D005786:Gene Expression Regulation / Q000502:physiology*; D015316:Genetic Therapy
chemical_list: D001426:Bacterial Proteins; D000076987:CRISPR-Associated Protein 9; C000606107:Cas9 endonuclease Streptococcus pyogenes; D004720:Endonucleases