CRISPR-derived genome editing therapies: Progress from bench to bedside

Mol Ther. 2021 Nov 3;29(11):3125-3139. doi: 10.1016/j.ymthe.2021.09.027. Epub 2021 Oct 5.

Abstract

The development of CRISPR-derived genome editing technologies has enabled the precise manipulation of DNA sequences within the human genome. In this review, we discuss the initial development and cellular mechanism of action of CRISPR nucleases and DNA base editors. We then describe factors that must be taken into consideration when developing these tools into therapeutic agents, including the potential for unintended and off-target edits when using these genome editing tools, and methods to characterize these types of edits. We finish by considering specific challenges associated with bringing a CRISPR-based therapy to the clinic, including manufacturing, regulatory oversight, and considerations for clinical trials that involve genome editing agents.

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Publication types

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

MeSH terms

  • Animals
  • CRISPR-Associated Protein 9
  • CRISPR-Cas Systems*
  • Clinical Trials as Topic
  • Clustered Regularly Interspaced Short Palindromic Repeats
  • Gene Editing* / methods
  • Gene Transfer Techniques
  • Genetic Engineering
  • Genetic Therapy* / methods
  • Genetic Therapy* / trends
  • Humans
  • Models, Animal
  • RNA, Guide, CRISPR-Cas Systems
  • Recombinational DNA Repair
  • Translational Research, Biomedical / methods
  • Translational Research, Biomedical / trends

Substances

  • RNA, Guide, CRISPR-Cas Systems
  • CRISPR-Associated Protein 9