Engineering Delivery Vehicles for Genome Editing

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...

Full description

Saved in:
Bibliographic Details
Published in:Annual review of chemical and biomolecular engineering 2016-06, Vol.7 (1), p.637-662
Main Authors: Nelson, Christopher E, Gersbach, Charles A
Format: Article
Language:English
Subjects:
Animals
CRISPR
CRISPR-Cas Systems - genetics
Editing
Fingers
Flexibility
Gene Editing - methods
Genetic modification
Genetic Therapy
Genetic Vectors - genetics
Genetic Vectors - metabolism
Genomes
Humans
Lentivirus - genetics
Liposomes - chemistry
Liposomes - metabolism
nanoparticle
nonviral gene therapy
TALENs
Transcription Activator-Like Effector Nucleases - genetics
Transcription Activator-Like Effector Nucleases - metabolism
Translations
Vehicles
viral gene therapy
virus
Zinc
zinc finger nucleases
Zinc Fingers - genetics
Citations: Items that this one cites
Items that cite this one
Online Access:Request full text
Tags: Add Tag
No Tags, Be the first to tag this record!
Description
Summary: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.
ISSN:1947-5438
1947-5446
DOI:10.1146/annurev-chembioeng-080615-034711