The current toolbox for APOBEC drug discovery

Michael J. Grillo, Katherine F.M. Jones, Michael A. Carpenter, Reuben S. Harris, Daniel A. Harki

Research output: Contribution to journalReview articlepeer-review

2 Scopus citations


Mutational processes driving genome evolution and heterogeneity contribute to immune evasion and therapy resistance in viral infections and cancer. APOBEC3 (A3) enzymes promote such mutations by catalyzing the deamination of cytosines to uracils in single-stranded DNA. Chemical inhibition of A3 enzymes may yield an antimutation therapeutic strategy to improve the durability of current drug therapies that are prone to resistance mutations. A3 small-molecule drug discovery efforts to date have been restricted to a single high-throughput biochemical activity assay; however, the arsenal of discovery assays has significantly expanded in recent years. The assays used to study A3 enzymes are reviewed here with an eye towards their potential for small-molecule discovery efforts.

Original languageEnglish (US)
Pages (from-to)362-377
Number of pages16
JournalTrends in Pharmacological Sciences
Issue number5
StatePublished - May 2022
Externally publishedYes


  • chemical probes
  • DNA deaminase inhibitors
  • drug discovery
  • screening

ASJC Scopus subject areas

  • Toxicology
  • Pharmacology


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