A chemical genetics approach to examine the functions of AAA proteins

Nat Struct Mol Biol. 2021 Apr;28(4):388-397. doi: 10.1038/s41594-021-00575-9. Epub 2021 Mar 29.

Abstract

The structural conservation across the AAA (ATPases associated with diverse cellular activities) protein family makes designing selective chemical inhibitors challenging. Here, we identify a triazolopyridine-based fragment that binds the AAA domain of human katanin, a microtubule-severing protein. We have developed a model for compound binding and designed ASPIR-1 (allele-specific, proximity-induced reactivity-based inhibitor-1), a cell-permeable compound that selectively inhibits katanin with an engineered cysteine mutation. Only in cells expressing mutant katanin does ASPIR-1 treatment increase the accumulation of CAMSAP2 at microtubule minus ends, confirming specific on-target cellular activity. Importantly, ASPIR-1 also selectively inhibits engineered cysteine mutants of human VPS4B and FIGL1-AAA proteins, involved in organelle dynamics and genome stability, respectively. Structural studies confirm our model for compound binding at the AAA ATPase site and the proximity-induced reactivity-based inhibition. Together, our findings suggest a chemical genetics approach to decipher AAA protein functions across essential cellular processes and to test hypotheses for developing therapeutics.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, Non-P.H.S.

MeSH terms

  • AAA Proteins / antagonists & inhibitors
  • AAA Proteins / genetics*
  • AAA Proteins / ultrastructure
  • ATPases Associated with Diverse Cellular Activities / genetics
  • ATPases Associated with Diverse Cellular Activities / ultrastructure
  • Basic Helix-Loop-Helix Transcription Factors / genetics
  • Endosomal Sorting Complexes Required for Transport / genetics
  • Endosomal Sorting Complexes Required for Transport / ultrastructure
  • Humans
  • Katanin / genetics*
  • Katanin / ultrastructure
  • Microtubule-Associated Proteins / genetics*
  • Microtubule-Associated Proteins / ultrastructure
  • Microtubules / genetics
  • Microtubules / ultrastructure
  • Protein Conformation / drug effects
  • Protein Domains / genetics
  • Pyridines / chemistry*
  • Pyridines / pharmacology
  • Triazoles / chemistry

Substances

  • Basic Helix-Loop-Helix Transcription Factors
  • CAMSAP2 protein, human
  • Endosomal Sorting Complexes Required for Transport
  • FIGLA protein, human
  • Microtubule-Associated Proteins
  • Pyridines
  • Triazoles
  • AAA Proteins
  • ATPases Associated with Diverse Cellular Activities
  • VPS4B protein, human
  • Katanin
  • pyridine