α-Helical peptidic scaffolds to target α-synuclein toxic species with nanomolar affinity

Nat Commun. 2021 Jun 18;12(1):3752. doi: 10.1038/s41467-021-24039-2.

Abstract

α-Synuclein aggregation is a key driver of neurodegeneration in Parkinson's disease and related syndromes. Accordingly, obtaining a molecule that targets α-synuclein toxic assemblies with high affinity is a long-pursued objective. Here, we exploit the biophysical properties of toxic oligomers and amyloid fibrils to identify a family of α-helical peptides that bind to these α-synuclein species with low nanomolar affinity, without interfering with the monomeric functional protein. This activity is translated into a high anti-aggregation potency and the ability to abrogate oligomer-induced cell damage. Using a structure-guided search we identify a human peptide expressed in the brain and the gastrointestinal tract with analogous binding, anti-aggregation, and detoxifying properties. The chemical entities we describe here may represent a therapeutic avenue for the synucleinopathies and are promising tools to assist diagnosis by discriminating between native and toxic α-synuclein species.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Amyloid / metabolism*
  • Brain / metabolism
  • Gastrointestinal Tract / metabolism
  • Humans
  • Parkinson Disease / pathology*
  • Protein Aggregation, Pathological / pathology*
  • alpha-Synuclein / metabolism*

Substances

  • Amyloid
  • alpha-Synuclein