A family with a mild form of congenital nystagmus and optic disc coloboma caused by a novel PAX6 mutation

Gene. 2019 Jul 15:705:177-180. doi: 10.1016/j.gene.2019.04.035. Epub 2019 Apr 12.

Abstract

Congenital nystagmus (CN) is a heterogeneous disease that shows variable clinical features. There are a few mutations that are known to cause CN. Among them, a PAX6 mutation is known to cause CN with an extremely high frequency of aniridia. Here, we report on a family with an autosomal dominant PAX6 mutation, c.214G > A (p.Gly72Ser.), who presented with CN in the absence of aniridia. This study describes detailed clinical findings, including videonystagmography and fundus photography findings and emphasizes the importance of screening for the PAX6 gene in patients who present with CN in the absence of aniridia, as this will further elucidate the known phenotypes of PAX6-related diseases.

MeSH terms

  • Child, Preschool
  • Coloboma / genetics
  • Coloboma / pathology*
  • Exome Sequencing / methods*
  • Female
  • Genetic Predisposition to Disease
  • Humans
  • Male
  • Nystagmus, Congenital / genetics
  • Nystagmus, Congenital / pathology*
  • Optic Disk / pathology
  • Optic Nerve / abnormalities*
  • Optic Nerve / pathology
  • PAX6 Transcription Factor / genetics*
  • Pedigree
  • Phenotype
  • Point Mutation*
  • Video Recording

Substances

  • PAX6 Transcription Factor
  • PAX6 protein, human

Supplementary concepts

  • Coloboma of optic nerve