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

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…

• MO
  Ministry of Science, ICT and Future Planning

  Award: 2015032163
• NH
  National Heart, Lung, and Blood Institute

  Award: HL109442
• NI
  National Institute of General Medical Sciences

  Award: GM110494
• NI
  National Institute of Allergy and Infectious Diseases

  Award: AI096305
• NC
  National Center for Advancing Translational Sciences

  Award: UH3TR000505