CRISPR challenges in clinical developments

Prog Mol Biol Transl Sci. 2025:210:263-279. doi: 10.1016/bs.pmbts.2024.08.001. Epub 2024 Aug 15.

Abstract

CRISPR-Cas (clustered regularly interspaced short palindromic repeats and associated proteins) is a novel genome editing technology with potential applications in treating diseases. Currently, its use in humans is restricted to clinical trials, although its growth rate is significant, and some have received initial FDA approval. It is crucial to examine and address the challenges for this technology to be implemented in clinical settings. This review aims to identify and explore new research ideas to increase of CRISPR's efficiency in treating genetic diseases and cancer, as well as its future prospects. Given that a substantial amount of previous research has focused on CRISPR-Cas delivery strategies and materials, this overview introduces specific conditions and strategies. It also discusses some of the challenges and opportunities in this field, offering a unique perspective.

Keywords: CRISPR-Cas9 system; Challenges; Clinical trial; Delivery systems; Off-target effects.

Publication types

  • Review

MeSH terms

  • Animals
  • CRISPR-Cas Systems* / genetics
  • Clustered Regularly Interspaced Short Palindromic Repeats* / genetics
  • Gene Editing
  • Humans
  • Neoplasms / genetics
  • Neoplasms / therapy