Restoring thalamocortical circuit dysfunction by correcting HCN channelopathy in Shank3 mutant mice

Cell Rep Med. 2024 May 21;5(5):101534. doi: 10.1016/j.xcrm.2024.101534. Epub 2024 Apr 25.

Abstract

Thalamocortical (TC) circuits are essential for sensory information processing. Clinical and preclinical studies of autism spectrum disorders (ASDs) have highlighted abnormal thalamic development and TC circuit dysfunction. However, mechanistic understanding of how TC dysfunction contributes to behavioral abnormalities in ASDs is limited. Here, our study on a Shank3 mouse model of ASD reveals TC neuron hyperexcitability with excessive burst firing and a temporal mismatch relationship with slow cortical rhythms during sleep. These TC electrophysiological alterations and the consequent sensory hypersensitivity and sleep fragmentation in Shank3 mutant mice are causally linked to HCN2 channelopathy. Restoring HCN2 function early in postnatal development via a viral approach or lamotrigine (LTG) ameliorates sensory and sleep problems. A retrospective case series also supports beneficial effects of LTG treatment on sensory behavior in ASD patients. Our study identifies a clinically relevant circuit mechanism and proposes a targeted molecular intervention for ASD-related behavioral impairments.

Keywords: ASDs; HCN2; Shank3; autism spectrum disorders; lamotrigine; neurodevelopmental disorders; sleep fragmentation; tactile hypersensitivity; thalamocortical circuits.

MeSH terms

  • Animals
  • Autism Spectrum Disorder* / genetics
  • Autism Spectrum Disorder* / metabolism
  • Autism Spectrum Disorder* / pathology
  • Autism Spectrum Disorder* / physiopathology
  • Cerebral Cortex / metabolism
  • Cerebral Cortex / pathology
  • Channelopathies / genetics
  • Channelopathies / metabolism
  • Channelopathies / pathology
  • Disease Models, Animal
  • Female
  • Humans
  • Hyperpolarization-Activated Cyclic Nucleotide-Gated Channels* / genetics
  • Hyperpolarization-Activated Cyclic Nucleotide-Gated Channels* / metabolism
  • Lamotrigine / pharmacology
  • Male
  • Mice
  • Mice, Inbred C57BL
  • Microfilament Proteins / genetics
  • Microfilament Proteins / metabolism
  • Mutation / genetics
  • Nerve Tissue Proteins* / genetics
  • Nerve Tissue Proteins* / metabolism
  • Neurons / metabolism
  • Potassium Channels
  • Sleep / drug effects
  • Sleep / genetics
  • Sleep / physiology
  • Thalamus* / metabolism
  • Thalamus* / pathology

Substances

  • Shank3 protein, mouse
  • Hyperpolarization-Activated Cyclic Nucleotide-Gated Channels
  • Nerve Tissue Proteins
  • Lamotrigine
  • Microfilament Proteins
  • Hcn2 protein, mouse
  • Potassium Channels