Login / Signup

Periventricular heterotopia in a male child with USP9X missense variant.

Arianna De LaurentiisClaudia CiaccioAlessandra ErbettaMichele PinelliVincenzo NigroChiara PantaleoniStefano D'Arrigo
Published in: American journal of medical genetics. Part A (2023)
The ubiquitin-specific protease USP9X has been found to play a role in multiple aspects of neural development including processes of neuronal migrations. In males, hemizygous partial loss of function variants in USP9X lead to a clinical phenotype primarily characterized by intellectual disability, hypotonia, speech and language impairment, behavioral disturbances accompanied by additional clinical features with variable expressivity. Structural brain abnormalities are reported in all cases where neuro-imaging was performed. The most common radiological features described include hypoplasia/agenesis of the corpus callosum, widened ventricles, white matter disturbances, and cerebellar hypoplasia. Here we report a child harboring a missense variant in USP9X presenting with the classical neurodevelopmental phenotype and a previously unreported radiological picture of periventricular heterotopia. This case expands the phenotypic landscape of this emergent condition and supports the critical role of USP9X in neuronal migration processes.
Keyphrases
  • intellectual disability
  • autism spectrum disorder
  • white matter
  • mental health
  • cerebral ischemia
  • multiple sclerosis
  • small molecule
  • case report
  • resting state
  • dna methylation
  • functional connectivity