Targeting notch signaling to restore neural development and behavior in mouse models of ASD

Nat Commun. 2026 Mar 30;17(1):2587. doi: 10.1038/s41467-026-70321-6.

Abstract

Autism spectrum disorder (ASD) is a neurodevelopmental disorder with diverse genetic and environmental origins, yet whether these factors converge on common molecular pathways remains unclear. This study identifies dysregulation of the Notch signaling pathway as a shared mechanism in both hereditary and nonhereditary ASD models. Aberrant histone deacetylase 3-mediated epigenetic regulation of Notch signaling during embryonic forebrain development disrupts the specification of vasoactive intestinal peptide (VIP + ) GABAergic interneuron subtypes (VIP-INs), which originate in the caudal ganglionic eminence (CGE). CGE-specific ablation of Notch1/2 genes in ASD models restores the loss of VIP-INs, normalizes maladaptive excitatory and inhibitory balance, and selectively improves social behaviors. A single antenatal dose of a γ-secretase inhibitor ameliorates multiple ASD-associated neuronal, behavioral, and transcriptomic changes in adult models. The study indicates a strong convergence of ASD-related factors on Notch signaling dysregulation and establishes this pathway as a promising therapeutic target for developmental and behavioral deficits in ASD.

MeSH terms

  • Amyloid Precursor Protein Secretases / antagonists & inhibitors
  • Amyloid Precursor Protein Secretases / metabolism
  • Animals
  • Autism Spectrum Disorder* / drug therapy
  • Autism Spectrum Disorder* / genetics
  • Autism Spectrum Disorder* / metabolism
  • Behavior, Animal / drug effects
  • Disease Models, Animal
  • Epigenesis, Genetic
  • Female
  • GABAergic Neurons / metabolism
  • Humans
  • Interneurons / metabolism
  • Male
  • Mice
  • Mice, Inbred C57BL
  • Neurogenesis / drug effects
  • Neurogenesis / genetics
  • Prosencephalon / embryology
  • Prosencephalon / metabolism
  • Receptor, Notch1* / genetics
  • Receptor, Notch1* / metabolism
  • Receptor, Notch2* / genetics
  • Receptor, Notch2* / metabolism
  • Receptors, Notch* / genetics
  • Receptors, Notch* / metabolism
  • Signal Transduction* / drug effects
  • Social Behavior

Substances

  • Receptor, Notch1
  • Receptor, Notch2
  • Notch1 protein, mouse
  • Notch2 protein, mouse
  • Receptors, Notch
  • Amyloid Precursor Protein Secretases