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CRISPR/Cas9-Based Genome Editing for Disease Modeling and Therapy: Challenges and Opportunities for Nonviral Delivery.

Publication ,  Journal Article
Wang, H-X; Li, M; Lee, CM; Chakraborty, S; Kim, H-W; Bao, G; Leong, KW
Published in: Chemical reviews
August 2017

Genome editing offers promising solutions to genetic disorders by editing DNA sequences or modulating gene expression. The clustered regularly interspaced short palindromic repeats (CRISPR)/associated protein 9 (CRISPR/Cas9) technology can be used to edit single or multiple genes in a wide variety of cell types and organisms in vitro and in vivo. Herein, we review the rapidly developing CRISPR/Cas9-based technologies for disease modeling and gene correction and recent progress toward Cas9/guide RNA (gRNA) delivery based on viral and nonviral vectors. We discuss the relative merits of delivering the genome editing elements in the form of DNA, mRNA, or protein, and the opportunities of combining viral delivery of a transgene encoding Cas9 with nonviral delivery of gRNA. We highlight the lessons learned from nonviral gene delivery in the past three decades and consider their applicability for CRISPR/Cas9 delivery. We also include a discussion of bioinformatics tools for gRNA design and chemical modifications of gRNA. Finally, we consider the extracellular and intracellular barriers to nonviral CRISPR/Cas9 delivery and propose strategies that may overcome these barriers to realize the clinical potential of CRISPR/Cas9-based genome editing.

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Published In

Chemical reviews

DOI

EISSN

1520-6890

ISSN

0009-2665

Publication Date

August 2017

Volume

117

Issue

15

Start / End Page

9874 / 9906

Related Subject Headings

  • Models, Biological
  • Humans
  • Genetic Therapy
  • General Chemistry
  • Gene Transfer Techniques
  • Gene Editing
  • CRISPR-Cas Systems
  • 40 Engineering
  • 34 Chemical sciences
  • 03 Chemical Sciences
 

Citation

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MLA
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Wang, H.-X., Li, M., Lee, C. M., Chakraborty, S., Kim, H.-W., Bao, G., & Leong, K. W. (2017). CRISPR/Cas9-Based Genome Editing for Disease Modeling and Therapy: Challenges and Opportunities for Nonviral Delivery. Chemical Reviews, 117(15), 9874–9906. https://doi.org/10.1021/acs.chemrev.6b00799
Wang, Hong-Xia, Mingqiang Li, Ciaran M. Lee, Syandan Chakraborty, Hae-Won Kim, Gang Bao, and Kam W. Leong. “CRISPR/Cas9-Based Genome Editing for Disease Modeling and Therapy: Challenges and Opportunities for Nonviral Delivery.Chemical Reviews 117, no. 15 (August 2017): 9874–9906. https://doi.org/10.1021/acs.chemrev.6b00799.
Wang H-X, Li M, Lee CM, Chakraborty S, Kim H-W, Bao G, et al. CRISPR/Cas9-Based Genome Editing for Disease Modeling and Therapy: Challenges and Opportunities for Nonviral Delivery. Chemical reviews. 2017 Aug;117(15):9874–906.
Wang, Hong-Xia, et al. “CRISPR/Cas9-Based Genome Editing for Disease Modeling and Therapy: Challenges and Opportunities for Nonviral Delivery.Chemical Reviews, vol. 117, no. 15, Aug. 2017, pp. 9874–906. Epmc, doi:10.1021/acs.chemrev.6b00799.
Wang H-X, Li M, Lee CM, Chakraborty S, Kim H-W, Bao G, Leong KW. CRISPR/Cas9-Based Genome Editing for Disease Modeling and Therapy: Challenges and Opportunities for Nonviral Delivery. Chemical reviews. 2017 Aug;117(15):9874–9906.
Journal cover image

Published In

Chemical reviews

DOI

EISSN

1520-6890

ISSN

0009-2665

Publication Date

August 2017

Volume

117

Issue

15

Start / End Page

9874 / 9906

Related Subject Headings

  • Models, Biological
  • Humans
  • Genetic Therapy
  • General Chemistry
  • Gene Transfer Techniques
  • Gene Editing
  • CRISPR-Cas Systems
  • 40 Engineering
  • 34 Chemical sciences
  • 03 Chemical Sciences