The Promise and Challenge of In Vivo Delivery for Genome Therapeutics

ACS Chem Biol. 2018 Feb 16;13(2):376-382. doi: 10.1021/acschembio.7b00680. Epub 2017 Oct 19.


CRISPR-based genome editing technologies are poised to enable countless new therapies to prevent, treat, or cure diseases with a genetic basis. However, the safe and effective delivery of genome editing enzymes represents a substantial challenge that must be tackled to enable the next generation of genetic therapies. In this Review, we summarize recent progress in developing enzymatic tools to combat genetic disease and examine current efforts to deliver these enzymes to the cells in need of correction. Viral vectors already in use for traditional gene therapy are being applied to enable in vivo CRISPR-based therapeutics, as are emerging technologies such as nanoparticle-based delivery of CRISPR components and direct delivery of preassembled RNA-protein complexes. Success in these areas will allow CRISPR-based genome editing therapeutics to reach their full potential.

Publication types

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

MeSH terms

  • Animals
  • CRISPR-Associated Proteins / genetics
  • CRISPR-Cas Systems / genetics*
  • Gene Editing / methods
  • Gene Transfer Techniques
  • Genetic Therapy / methods*
  • Genetic Vectors / genetics
  • Genome / genetics*
  • Humans
  • Nanoparticles / chemistry
  • Ribonucleoproteins / genetics
  • Viruses / genetics


  • CRISPR-Associated Proteins
  • Ribonucleoproteins