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Engineering Delivery Vehicles for Genome Editing.

Publication ,  Journal Article
Nelson, CE; Gersbach, CA
Published in: Annual review of chemical and biomolecular engineering
June 2016

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.

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Published In

Annual review of chemical and biomolecular engineering

DOI

EISSN

1947-5446

ISSN

1947-5438

Publication Date

June 2016

Volume

7

Start / End Page

637 / 662

Related Subject Headings

  • Zinc Fingers
  • Transcription Activator-Like Effector Nucleases
  • Liposomes
  • Lentivirus
  • Humans
  • Genetic Vectors
  • Genetic Therapy
  • Gene Editing
  • CRISPR-Cas Systems
  • Animals
 

Citation

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Nelson, C. E., & Gersbach, C. A. (2016). Engineering Delivery Vehicles for Genome Editing. Annual Review of Chemical and Biomolecular Engineering, 7, 637–662. https://doi.org/10.1146/annurev-chembioeng-080615-034711
Nelson, Christopher E., and Charles A. Gersbach. “Engineering Delivery Vehicles for Genome Editing.Annual Review of Chemical and Biomolecular Engineering 7 (June 2016): 637–62. https://doi.org/10.1146/annurev-chembioeng-080615-034711.
Nelson CE, Gersbach CA. Engineering Delivery Vehicles for Genome Editing. Annual review of chemical and biomolecular engineering. 2016 Jun;7:637–62.
Nelson, Christopher E., and Charles A. Gersbach. “Engineering Delivery Vehicles for Genome Editing.Annual Review of Chemical and Biomolecular Engineering, vol. 7, June 2016, pp. 637–62. Epmc, doi:10.1146/annurev-chembioeng-080615-034711.
Nelson CE, Gersbach CA. Engineering Delivery Vehicles for Genome Editing. Annual review of chemical and biomolecular engineering. 2016 Jun;7:637–662.

Published In

Annual review of chemical and biomolecular engineering

DOI

EISSN

1947-5446

ISSN

1947-5438

Publication Date

June 2016

Volume

7

Start / End Page

637 / 662

Related Subject Headings

  • Zinc Fingers
  • Transcription Activator-Like Effector Nucleases
  • Liposomes
  • Lentivirus
  • Humans
  • Genetic Vectors
  • Genetic Therapy
  • Gene Editing
  • CRISPR-Cas Systems
  • Animals