Sequential CRISPR screening reveals partial NatB inhibition as a strategy to mitigate alpha-synuclein levels in human neurons

Sci Adv. 2024 Feb 9;10(6):eadj4767. doi: 10.1126/sciadv.adj4767. Epub 2024 Feb 9.

Abstract

Alpha-synuclein (αSyn) protein levels correlate with the risk and severity of Parkinson's disease and related neurodegenerative diseases. Lowering αSyn is being actively investigated as a therapeutic modality. Here, we systematically map the regulatory network that controls endogenous αSyn using sequential CRISPR-knockout and -interference screens in an αSyn gene (SNCA)-tagged cell line and induced pluripotent stem cell-derived neurons (iNeurons). We uncover αSyn modifiers at multiple regulatory layers, with amino-terminal acetyltransferase B (NatB) enzymes being the most potent endogenous αSyn modifiers in both cell lines. Amino-terminal acetylation protects the cytosolic αSyn from rapid degradation by the proteasome in a Ube2w-dependent manner. Moreover, we show that pharmacological inhibition of methionyl-aminopeptidase 2, a regulator of NatB complex formation, attenuates endogenous αSyn in iNeurons carrying SNCA triplication. Together, our study reveals several gene networks that control endogenous αSyn, identifies mechanisms mediating the degradation of nonacetylated αSyn, and illustrates potential therapeutic pathways for decreasing αSyn levels in synucleinopathies.

MeSH terms

  • Cell Line
  • Clustered Regularly Interspaced Short Palindromic Repeats
  • Humans
  • Methionyl Aminopeptidases / antagonists & inhibitors
  • Methionyl Aminopeptidases / metabolism
  • N-Terminal Acetyltransferase B* / antagonists & inhibitors
  • N-Terminal Acetyltransferase B* / metabolism
  • Neurons / metabolism
  • Parkinson Disease* / genetics
  • Parkinson Disease* / metabolism
  • alpha-Synuclein* / genetics
  • alpha-Synuclein* / metabolism

Substances

  • alpha-Synuclein
  • NAA20 protein, human
  • N-Terminal Acetyltransferase B
  • METAP2 protein, human
  • Methionyl Aminopeptidases