PEX1 deficiency presenting as Leber congenital amaurosis

Pediatr Neurol. 2004 Aug;31(2):146-9. doi: 10.1016/j.pediatrneurol.2004.01.013.

Abstract

Peroxisome biogenesis disorders result from defects in peroxin proteins involved in peroxisomal matrix and membrane protein import. Peroxins are encoded in peroxin protein genes; to date, the PEX genes responsible for all 12 peroxisome biogenesis disorders complementation groups are known. Peroxin protein 1 deficiency associated with complementation group 1 is responsible for disease in approximately two thirds of all patients with a peroxisome biogenesis disorder. Their phenotypes range from severe to mild, and it appears to be a phenotype-genotype relationship. This case report describes a patient with peroxin protein 1 deficiency presenting as Leber congenital amaurosis, in whom the diagnosis was questioned at the age of 2 years when seizures first appeared and mild facial dysmorphia became evident.

Publication types

  • Case Reports

MeSH terms

  • ATPases Associated with Diverse Cellular Activities
  • Child, Preschool
  • Diagnosis, Differential
  • Female
  • Humans
  • Magnetic Resonance Imaging / methods
  • Membrane Proteins / deficiency*
  • Optic Atrophy, Hereditary, Leber / diagnosis*
  • Optic Atrophy, Hereditary, Leber / metabolism*

Substances

  • Membrane Proteins
  • ATPases Associated with Diverse Cellular Activities
  • PEX1 protein, human