Autosomal recessive retinal dystrophy associated with two novel mutations in the RPE65 gene

Eur J Hum Genet. 1998 Sep-Oct;6(5):527-31. doi: 10.1038/sj.ejhg.5200205.

Abstract

Retinal dystrophies are a complex set of hereditary diseases of the retina that result in the degeneration of photoreceptors. Recent studies have shown that mutations in RPE65, a gene that codes for a retinal pigment epithelium (RPE)-specific protein thought to be involved in the 11-cis-retinoid metabolism, a key process in vision, cause severe, early onset retinal dystrophy. We describe two novel missense RPE65 mutations, L22P and H68Y, in a compound heterozygote with autosomal recessive retinal dystrophy. The relatively mild phenotype associated with these mutations suggests a possible link between the severity of the disease and the type of mutations in the RPE65 gene.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Amino Acid Sequence
  • Amino Acid Substitution
  • Base Sequence
  • Carrier Proteins
  • DNA
  • Eye Proteins / genetics*
  • Female
  • Genes, Recessive*
  • Heterozygote
  • Humans
  • Male
  • Mutation*
  • Pedigree
  • Pigment Epithelium of Eye / metabolism*
  • Proteins*
  • Retinal Diseases / genetics*
  • cis-trans-Isomerases

Substances

  • Carrier Proteins
  • Eye Proteins
  • Proteins
  • DNA
  • retinoid isomerohydrolase
  • cis-trans-Isomerases