Engineering Delivery Vehicles for Genome Editing

TitleEngineering Delivery Vehicles for Genome Editing
Publication TypeJournal Article
Year of Publication2016
AuthorsNelson, CE, and Gersbach, CA
JournalAnnual Review of Chemical and Biomolecular Engineering
Volume7
Issue1
Start Page637
Pagination637 - 662
Date Published06/2016
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.

DOI10.1146/annurev-chembioeng-080615-034711
Short TitleAnnual Review of Chemical and Biomolecular Engineering