Nanoparticle-Based Delivery of CRISPR/Cas9 Genome-Editing Therapeutics

AAPS J. 2018 Oct 10;20(6):108. doi: 10.1208/s12248-018-0267-9.

Abstract

The recent progress in harnessing the efficient and precise method of DNA editing provided by CRISPR/Cas9 is one of the most promising major advances in the field of gene therapy. However, the development of safe and optimally efficient delivery systems for CRISPR/Cas9 elements capable of achieving specific targeting of gene therapy to the location of interest without off-target effects is a primary challenge for clinical therapeutics. Nanoparticles (NPs) provide a promising means to meet such challenges. In this review, we present the most recent advances in developing innovative NP-based delivery systems that efficiently deliver CRISPR/Cas9 constructs and maximize their effectiveness.

Keywords: CRISPR/Cas9; gene delivery; gene editing; nanoparticle.

Publication types

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

MeSH terms

  • CRISPR-Cas Systems / genetics*
  • DNA / genetics
  • Gene Editing / methods*
  • Gene Transfer Techniques*
  • Genetic Therapy / methods*
  • Humans
  • Nanoparticles / chemistry*

Substances

  • DNA