title: Engineering Delivery Vehicles for Genome Editing.
abstract: The field of genome engineering has created new possibilities for gene therapy, including improved animal models of disease, engineered cell therapies, and in vivo gene repair. The most significant challenge for the clinical translation of genome engineering is the development of safe and effective delivery vehicles. A large body of work has applied genome engineering to genetic modification in vitro, and clinical trials have begun using cells modified by genome editing. Now, promising preclinical work is beginning to apply these tools in vivo. This article summarizes the development of genome engineering platforms, including meganucleases, zinc finger nucleases, TALENs, and CRISPR/Cas9, and their flexibility for precise genetic modifications. The prospects for the development of safe and effective viral and nonviral delivery vehicles for genome editing are reviewed, and promising advances in particular therapeutic applications are discussed.
authors.affiliation: Department of Biomedical Engineering, Duke University, Durham, North Carolina 27708.|Center for Genomic & Computational Biology, Duke University, Durham, North Carolina 27708.
authors.forename: Christopher E
authors.initials: CE
authors.lastname: Nelson
authors.affiliation: Department of Biomedical Engineering, Duke University, Durham, North Carolina 27708.|Center for Genomic & Computational Biology, Duke University, Durham, North Carolina 27708.|Department of Orthopaedic Surgery, Duke University, Durham, North Carolina 27708; email: charles.gersbach@duke.edu.
authors.forename: Charles A
authors.initials: CA
authors.lastname: Gersbach
mesh_terms: D000818:Animals; D064113:CRISPR-Cas Systems / Q000235:genetics; D000072669:Gene Editing / Q000379:methods*; D015316:Genetic Therapy; D005822:Genetic Vectors / Q000235:genetics / Q000378:metabolism; D006801:Humans; D016086:Lentivirus / Q000235:genetics; D008081:Liposomes / Q000737:chemistry / Q000378:metabolism; D000069896:Transcription Activator-Like Effector Nucleases / Q000235:genetics / Q000378:metabolism; D016335:Zinc Fingers / Q000235:genetics
chemical_list: D008081:Liposomes; D000069896:Transcription Activator-Like Effector Nucleases
keywords: CRISPR; TALENs; nanoparticle; nonviral gene therapy; viral gene therapy; virus; zinc finger nucleases