Intrafamilial clinical heterogeneity associated with a novel mutation of the retinal degeneration slow/peripherin gene

Ophthalmic Res. 2007;39(5):255-9. doi: 10.1159/000108118. Epub 2007 Sep 12.

Abstract

Aims: To identify the phenotypic variations in 6 related individuals affected by a novel mutation in the retinal degeneration slow/peripherin gene.

Methods: Ten family members underwent ophthalmologic assessment with slit-lamp biomicroscopy, dilated fundus examination, fundus photography, autofluorescence imaging and electrophysiological tests. Genomic DNA was extracted from blood samples of all family members (n = 15) using the standard salting-out procedure.

Results: The novel C165R mutation was identified in 8 individuals. Of these 8 patients, only 6 gave consent to the clinical study. They had a retinal disease characterized by an adulthood onset of symptoms, and their best corrected visual acuity was between 20/50 and 20/20. Fundus examination showed that 3 patients had typical fundus flavimaculatus: 1 had butterfly-shaped pattern dystrophy and 2 had incipient retinal changes.

Conclusion: We identified a novel mutation of the retinal degeneration slow/peripherin gene in a family affected by different patterns of retinal dystrophy. This is the first report of an association of fundus flavimaculatus with butterfly-shaped pattern dystrophy.

MeSH terms

  • Aged
  • Arginine
  • Cysteine
  • Electrooculography
  • Electroretinography
  • Fluorescence
  • Fundus Oculi
  • Genetic Heterogeneity
  • Humans
  • Intermediate Filament Proteins / genetics*
  • Membrane Glycoproteins / genetics*
  • Middle Aged
  • Mutation*
  • Nerve Tissue Proteins / genetics*
  • Pedigree
  • Peripherins
  • Phenotype
  • Retinal Degeneration / diagnosis
  • Retinal Degeneration / genetics*
  • Retinal Degeneration / physiopathology
  • Visual Acuity

Substances

  • Intermediate Filament Proteins
  • Membrane Glycoproteins
  • Nerve Tissue Proteins
  • PRPH protein, human
  • Peripherins
  • Arginine
  • Cysteine