title: Genome-editing Technologies for Gene and Cell Therapy.
abstract: Gene therapy has historically been defined as the addition of new genes to human cells. However, the recent advent of genome-editing technologies has enabled a new paradigm in which the sequence of the human genome can be precisely manipulated to achieve a therapeutic effect. This includes the correction of mutations that cause disease, the addition of therapeutic genes to specific sites in the genome, and the removal of deleterious genes or genome sequences. This review presents the mechanisms of different genome-editing strategies and describes each of the common nuclease-based platforms, including zinc finger nucleases, transcription activator-like effector nucleases (TALENs), meganucleases, and the CRISPR/Cas9 system. We then summarize the progress made in applying genome editing to various areas of gene and cell therapy, including antiviral strategies, immunotherapies, and the treatment of monogenic hereditary disorders. The current challenges and future prospects for genome editing as a transformative technology for gene and cell therapy are also discussed.
authors.affiliation: Editas Medicine, Cambridge, Massachusetts, USA.
authors.forename: Morgan L
authors.initials: ML
authors.lastname: Maeder
authors.affiliation: Department of Biomedical Engineering, Duke University, Durham, North Carolina, USA.|Center for Genomic and Computational Biology, Duke University, Durham, North Carolina, USA.|Department of Orthopaedic Surgery, Duke University Medical Center, Durham, North Carolina, USA.
authors.forename: Charles A
authors.initials: CA
authors.lastname: Gersbach
mesh_terms: D000818:Animals; D064987:Cell- and Tissue-Based Therapy / Q000379:methods; D017321:Clinical Trials, Phase I as Topic; D017322:Clinical Trials, Phase II as Topic; D004353:Drug Evaluation, Preclinical; D004720:Endonucleases / Q000378:metabolism; D000072669:Gene Editing / Q000379:methods*; D018390:Gene Targeting / Q000379:methods; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods; D005822:Genetic Vectors / Q000235:genetics; D016678:Genome; D006801:Humans
chemical_list: D004720:Endonucleases