Hide-and-Seek genome editing reveals that Gephyrin is required for axo-axonic synapse assembly

Proc Natl Acad Sci U S A. 2025 Aug 12;122(32):e2500726122. doi: 10.1073/pnas.2500726122. Epub 2025 Aug 7.

Abstract

The visualization and manipulation of proteins in neurons is widely used to deduce their functions. While every experimental approach has limitations, the concurrent knock-in and knockout of two different proteins can be especially challenging. To this end, we developed Hide-and-Seek genome editing, which allows the simultaneous visualization and knockout of proteins in neurons using Adeno-associated viral vectors and the CRISPR/Cas9 system. We demonstrate the efficacy and flexibility of this method for rapid, efficient, and simultaneous knock-in and knockout of proteins in vitro and in vivo, at the synapse, axon initial segment (AIS), nucleus, and mitochondria. Using Hide-and-Seek, we show that the scaffolding protein Gephyrin is required for the proper assembly of axo-axonic synapses at the AIS.

Keywords: AAV; CRISPR; axon; axon initial segment; inhibitory synapse.

MeSH terms

  • Animals
  • Axons* / metabolism
  • CRISPR-Cas Systems
  • Carrier Proteins* / genetics
  • Carrier Proteins* / metabolism
  • Dependovirus / genetics
  • Gene Editing* / methods
  • Membrane Proteins* / genetics
  • Membrane Proteins* / metabolism
  • Mice
  • Mitochondria / metabolism
  • Neurons / metabolism
  • Synapses* / genetics
  • Synapses* / metabolism

Substances

  • Membrane Proteins
  • gephyrin
  • Carrier Proteins