Engineering Delivery Vehicles for Genome Editing.

Journal Article (Review;Journal Article)

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.

Full Text

Duke Authors

Cited Authors

  • Nelson, CE; Gersbach, CA

Published Date

  • June 2016

Published In

Volume / Issue

  • 7 /

Start / End Page

  • 637 - 662

PubMed ID

  • 27146557

Electronic International Standard Serial Number (EISSN)

  • 1947-5446

International Standard Serial Number (ISSN)

  • 1947-5438

Digital Object Identifier (DOI)

  • 10.1146/annurev-chembioeng-080615-034711


  • eng