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Genome-editing Technologies for Gene and Cell Therapy.

Publication ,  Journal Article
Maeder, ML; Gersbach, CA
Published in: Molecular therapy : the journal of the American Society of Gene Therapy
March 2016

Gene therapy has historically been defined as the addition of new genes to human cells. However, the recent advent of genome-editing technologies has enabled a new paradigm in which the sequence of the human genome can be precisely manipulated to achieve a therapeutic effect. This includes the correction of mutations that cause disease, the addition of therapeutic genes to specific sites in the genome, and the removal of deleterious genes or genome sequences. This review presents the mechanisms of different genome-editing strategies and describes each of the common nuclease-based platforms, including zinc finger nucleases, transcription activator-like effector nucleases (TALENs), meganucleases, and the CRISPR/Cas9 system. We then summarize the progress made in applying genome editing to various areas of gene and cell therapy, including antiviral strategies, immunotherapies, and the treatment of monogenic hereditary disorders. The current challenges and future prospects for genome editing as a transformative technology for gene and cell therapy are also discussed.

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Published In

Molecular therapy : the journal of the American Society of Gene Therapy

DOI

EISSN

1525-0024

ISSN

1525-0016

Publication Date

March 2016

Volume

24

Issue

3

Start / End Page

430 / 446

Related Subject Headings

  • Humans
  • Genome
  • Genetic Vectors
  • Genetic Therapy
  • Gene Transfer Techniques
  • Gene Targeting
  • Gene Editing
  • Endonucleases
  • Drug Evaluation, Preclinical
  • Clinical Trials, Phase II as Topic
 

Citation

APA
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ICMJE
MLA
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Maeder, M. L., & Gersbach, C. A. (2016). Genome-editing Technologies for Gene and Cell Therapy. Molecular Therapy : The Journal of the American Society of Gene Therapy, 24(3), 430–446. https://doi.org/10.1038/mt.2016.10
Maeder, Morgan L., and Charles A. Gersbach. “Genome-editing Technologies for Gene and Cell Therapy.Molecular Therapy : The Journal of the American Society of Gene Therapy 24, no. 3 (March 2016): 430–46. https://doi.org/10.1038/mt.2016.10.
Maeder ML, Gersbach CA. Genome-editing Technologies for Gene and Cell Therapy. Molecular therapy : the journal of the American Society of Gene Therapy. 2016 Mar;24(3):430–46.
Maeder, Morgan L., and Charles A. Gersbach. “Genome-editing Technologies for Gene and Cell Therapy.Molecular Therapy : The Journal of the American Society of Gene Therapy, vol. 24, no. 3, Mar. 2016, pp. 430–46. Epmc, doi:10.1038/mt.2016.10.
Maeder ML, Gersbach CA. Genome-editing Technologies for Gene and Cell Therapy. Molecular therapy : the journal of the American Society of Gene Therapy. 2016 Mar;24(3):430–446.

Published In

Molecular therapy : the journal of the American Society of Gene Therapy

DOI

EISSN

1525-0024

ISSN

1525-0016

Publication Date

March 2016

Volume

24

Issue

3

Start / End Page

430 / 446

Related Subject Headings

  • Humans
  • Genome
  • Genetic Vectors
  • Genetic Therapy
  • Gene Transfer Techniques
  • Gene Targeting
  • Gene Editing
  • Endonucleases
  • Drug Evaluation, Preclinical
  • Clinical Trials, Phase II as Topic