PARG inhibition induces nuclear aggregation of PARylated PARP1

Structure. 2024 Nov 7;32(11):2083-2093.e5. doi: 10.1016/j.str.2024.09.006. Epub 2024 Oct 14.

Abstract

Poly (ADP-ribose) glycohydrolase (PARG) inhibitors are currently under clinical development for the treatment of DNA repair-deficient cancers; however, their precise mechanism of action is still unclear. Here, we report that PARG inhibition leads to excessive PARylated poly (ADP-ribose) polymerase 1 (PARP1) reducing the ability of PARP1 to properly localize to sites of DNA damage. Strikingly, the mis-localized PARP1 accumulates as aggregates throughout the nucleus. Abrogation of the catalytic activity of PARP1 prevents aggregate formation, indicating that PAR chains play a key role in this process. Finally, we find that PARP1 nuclear aggregates were highly persistent and were associated with cleaved cytoplasmic PARP1, ultimately leading to cell death. Overall, our data uncover an unexpected mechanism of PARG inhibitor cytotoxicity, which will shed light on the use of these drugs as anti-cancer therapeutics.

Keywords: PAR; PARG; PARG inhibitors; PARP1; PARP1 aggregation; laser microirradiation.

MeSH terms

  • Cell Nucleus* / metabolism
  • DNA Damage
  • Enzyme Inhibitors / chemistry
  • Enzyme Inhibitors / pharmacology
  • Glycoside Hydrolases* / chemistry
  • Glycoside Hydrolases* / genetics
  • Glycoside Hydrolases* / metabolism
  • Humans
  • Poly (ADP-Ribose) Polymerase-1* / antagonists & inhibitors
  • Poly (ADP-Ribose) Polymerase-1* / genetics
  • Poly (ADP-Ribose) Polymerase-1* / metabolism
  • Poly ADP Ribosylation
  • Poly(ADP-ribose) Polymerase Inhibitors / pharmacology
  • Protein Aggregates

Substances

  • poly ADP-ribose glycohydrolase
  • Poly (ADP-Ribose) Polymerase-1
  • PARP1 protein, human
  • Glycoside Hydrolases
  • Enzyme Inhibitors
  • Protein Aggregates
  • Poly(ADP-ribose) Polymerase Inhibitors