In Vivo Delivery of CRISPR/Cas9 for Therapeutic Gene Editing: Progress and Challenges

Bioconjug Chem. 2017 Apr 19;28(4):880-884. doi: 10.1021/acs.bioconjchem.7b00057. Epub 2017 Mar 17.

Abstract

The successful use of clustered regularly interspaced short palindromic repeat (CRISPR)/Cas9-based gene editing for therapeutics requires efficient in vivo delivery of the CRISPR components. There are, however, major challenges on the delivery front. In this Topical Review, we will highlight recent developments in CRISPR delivery, and we will present hurdles that still need to be overcome to achieve effective in vivo editing.

Publication types

  • Review

MeSH terms

  • Animals
  • CRISPR-Cas Systems*
  • Clustered Regularly Interspaced Short Palindromic Repeats*
  • Gene Editing / methods*
  • Gene Transfer Techniques
  • Genetic Therapy / methods
  • Genetic Vectors / administration & dosage
  • Genetic Vectors / genetics
  • Humans
  • Mutagenesis, Insertional / methods
  • Viruses / genetics