Missense mutations in a retinal pigment epithelium protein, bestrophin-1, cause retinitis pigmentosa

Am J Hum Genet. 2009 Nov;85(5):581-92. doi: 10.1016/j.ajhg.2009.09.015. Epub 2009 Oct 22.

Abstract

Bestrophin-1 is preferentially expressed at the basolateral membrane of the retinal pigmented epithelium (RPE) of the retina. Mutations in the BEST1 gene cause the retinal dystrophies vitelliform macular dystrophy, autosomal-dominant vitreochoroidopathy, and autosomal-recessive bestrophinopathy. Here, we describe four missense mutations in bestrophin-1, three that we believe are previously unreported, in patients diagnosed with autosomal-dominant and -recessive forms of retinitis pigmentosa (RP). The physiological function of bestrophin-1 remains poorly understood although its heterologous expression induces a Cl--specific current. We tested the effect of RP-causing variants on Cl- channel activity and cellular localization of bestrophin-1. Two (p.L140V and p.I205T) produced significantly decreased chloride-selective whole-cell currents in comparison to those of wild-type protein. In a model system of a polarized epithelium, two of three mutations (p.L140V and p.D228N) caused mislocalization of bestrophin-1 from the basolateral membrane to the cytoplasm. Mutations in bestrophin-1 are increasingly recognized as an important cause of inherited retinal dystrophy.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Amino Acid Sequence
  • Bestrophins
  • Cell Line
  • Chloride Channels / genetics*
  • Chromosomes, Human, Pair 11
  • Conserved Sequence
  • Exons
  • Eye Proteins / genetics*
  • Female
  • Genes, Dominant
  • Genes, Recessive
  • Genetic Linkage
  • Homozygote
  • Humans
  • Kidney / cytology
  • Lod Score
  • Male
  • Molecular Sequence Data
  • Mutation, Missense*
  • Nuclear Family
  • Pedigree
  • Polymorphism, Single Nucleotide
  • Protein Isoforms / genetics
  • Protein Isoforms / metabolism
  • Retinal Pigment Epithelium / pathology*
  • Retinitis Pigmentosa / etiology*
  • Retinitis Pigmentosa / genetics*
  • Sequence Homology, Amino Acid

Substances

  • BEST1 protein, human
  • Bestrophins
  • Chloride Channels
  • Eye Proteins
  • Protein Isoforms