Engineering the Delivery System for CRISPR-Based Genome Editing

Trends Biotechnol. 2018 Feb;36(2):173-185. doi: 10.1016/j.tibtech.2017.11.006. Epub 2018 Jan 2.

Abstract

Clustered regularly interspaced short palindromic repeat-CRISPR-associated protein (CRISPR-Cas) systems, found in nature as microbial adaptive immune systems, have been repurposed into an important tool in biological engineering and genome editing, providing a programmable platform for precision gene targeting. These tools have immense promise as therapeutics that could potentially correct disease-causing mutations. However, CRISPR-Cas gene editing components must be transported directly to the nucleus of targeted cells to exert a therapeutic effect. Thus, efficient methods of delivery will be critical to the success of therapeutic genome editing applications. Here, we review current strategies available for in vivo delivery of CRISPR-Cas gene editing components and outline challenges that need to be addressed before this powerful tool can be deployed in the clinic.

Keywords: CRISPR; Cas9; clinical; delivery; gene editing; therapeutics.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, U.S. Gov't, Non-P.H.S.
  • Review

MeSH terms

  • Bacterial Proteins / genetics
  • Bacterial Proteins / metabolism
  • CRISPR-Associated Protein 9
  • CRISPR-Cas Systems*
  • Dependovirus / genetics*
  • Dependovirus / metabolism
  • Electroporation / methods
  • Endonucleases / genetics
  • Endonucleases / metabolism
  • Gene Editing / methods*
  • Gene Targeting / methods
  • Gene Transfer Techniques*
  • Genetic Engineering / methods
  • Genetic Vectors / chemistry*
  • Genetic Vectors / metabolism
  • Humans
  • Microinjections / methods
  • Nanoparticles / administration & dosage
  • Plasmids / chemistry
  • Plasmids / metabolism
  • RNA, Guide, CRISPR-Cas Systems / genetics
  • RNA, Guide, CRISPR-Cas Systems / metabolism

Substances

  • Bacterial Proteins
  • RNA, Guide, CRISPR-Cas Systems
  • CRISPR-Associated Protein 9
  • Cas9 endonuclease Streptococcus pyogenes
  • Endonucleases