Synergistic inhibition of histone modifiers produces therapeutic effects in adult Shank3-deficient mice

Transl Psychiatry. 2021 Feb 4;11(1):99. doi: 10.1038/s41398-021-01233-w.

Abstract

Autism spectrum disorder (ASD) is a lifelong developmental disorder characterized by social deficits and other behavioral abnormalities. Dysregulation of epigenetic processes, such as histone modifications and chromatin remodeling, have been implicated in ASD pathology, and provides a promising therapeutic target for ASD. Haploinsufficiency of the SHANK3 gene is causally linked to ASD, so adult (3-5 months old) Shank3-deficient male mice were used in this drug discovery study. We found that combined administration of the class I histone deacetylase inhibitor Romidepsin and the histone demethylase LSD1 inhibitor GSK-LSD1 persistently ameliorated the autism-like social preference deficits, while each individual drug alone was largely ineffective. Another behavioral abnormality in adult Shank3-deficient male mice, heightened aggression, was also alleviated by administration of the dual drugs. Furthermore, Romidepsin/GSK-LSD1 treatment significantly increased transcriptional levels of NMDA receptor subunits in prefrontal cortex (PFC) of adult Shank3-deficient mice, resulting in elevated synaptic expression of NMDA receptors and the restoration of NMDAR synaptic function in PFC pyramidal neurons. These results have offered a novel pharmacological intervention strategy for ASD beyond early developmental periods.

Publication types

  • Research Support, N.I.H., Extramural

MeSH terms

  • Animals
  • Autism Spectrum Disorder* / drug therapy
  • Autism Spectrum Disorder* / genetics
  • Disease Models, Animal
  • Histones*
  • Male
  • Mice
  • Microfilament Proteins
  • Nerve Tissue Proteins / metabolism

Substances

  • Histones
  • Microfilament Proteins
  • Nerve Tissue Proteins
  • Shank3 protein, mouse