Small nucleic acids and the path to the clinic for anti-CRISPR

Christopher L. Barkau, Daniel O'Reilly, Seth B. Eddington, Masad J. Damha, Keith T. Gagnon

Research output: Contribution to journalReview articlepeer-review

8 Scopus citations

Abstract

CRISPR-based therapeutics have entered clinical trials but no methods to inhibit Cas enzymes have been demonstrated in a clinical setting. The ability to inhibit CRISPR-based gene editing or gene targeting drugs should be considered a critical step in establishing safety standards for many CRISPR-Cas therapeutics. Inhibitors can act as a failsafe or as an adjuvant to reduce off-target effects in patients. In this review we discuss the need for clinical inhibition of CRISPR-Cas systems and three existing inhibitor technologies: anti-CRISPR (Acr) proteins, small molecule Cas inhibitors, and small nucleic acid-based CRISPR inhibitors, CRISPR SNuBs. Due to their unique properties and the recent successes of other nucleic acid-based therapeutics, CRISPR SNuBs appear poised for clinical application in the near-term.

Original languageEnglish (US)
Article number114492
JournalBiochemical Pharmacology
Volume189
DOIs
StatePublished - Jul 2021
Externally publishedYes

ASJC Scopus subject areas

  • Biochemistry
  • Pharmacology

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