Nonviral Nanoparticles for CRISPR-Based Genome Editing: Is It Just a Simple Adaption of What Have Been Developed for Nucleic Acid Delivery?

Biomacromolecules. 2019 Sep 9;20(9):3333-3339. doi: 10.1021/acs.biomac.9b00783. Epub 2019 Aug 7.


Genome-editing technologies hold tremendous potential for treating genetic diseases. However, the efficient and safe delivery of genome-editing elements to the location of interest, and the achievement of specific targeted gene correction without off-target side effect remains a big challenge. In this Perspective, we highlight recent developments and discuss the challenges of nonviral nanoparticles for the delivery of genome-editing tools. Finally, we will propose promising strategies to improve the delivery efficacy and advance the clinical translation of gene-editing technology.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • CRISPR-Cas Systems / genetics*
  • Gene Editing / methods
  • Gene Transfer Techniques*
  • Genetic Therapy
  • Humans
  • Nanoparticles / chemistry*
  • Nanoparticles / therapeutic use
  • Nucleic Acids / genetics*
  • Nucleic Acids / therapeutic use


  • Nucleic Acids