Dominant Stargardt Macular Dystrophy (STGD3) and ELOVL4

Adv Exp Med Biol. 2014:801:447-53. doi: 10.1007/978-1-4614-3209-8_57.

Abstract

Autosomal dominant Stargardt3 Macular Dystrophy (STGD3) results from mutations in the ELOVL4 gene. ELOVL4 protein localizes to the endoplasmic reticulum (ER), where it mediates the rate-limiting condensation reaction during very long-chain (VLC, ≥ C28) fatty acid biosynthesis. The defective gene product is truncated at the C-terminus, leading to mislocalization and aggregation in other organelles. In this review, we summarize our current understanding of the disease-causing mutation and its potential role in STGD3 pathogenesis.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Endoplasmic Reticulum / metabolism
  • Eye Proteins / genetics*
  • Eye Proteins / metabolism
  • Genes, Dominant
  • Humans
  • Macular Degeneration / congenital*
  • Macular Degeneration / genetics
  • Macular Degeneration / metabolism
  • Macular Degeneration / physiopathology
  • Membrane Proteins / genetics*
  • Membrane Proteins / metabolism

Substances

  • ELOVL4 protein, human
  • Eye Proteins
  • Membrane Proteins

Supplementary concepts

  • Stargardt disease 3