Mutations in the phospholipid remodeling gene SERAC1 impair mitochondrial function and intracellular cholesterol trafficking and cause dystonia and deafness

Nat Genet. 2012 Jun 10;44(7):797-802. doi: 10.1038/ng.2325.

Abstract

Using exome sequencing, we identify SERAC1 mutations as the cause of MEGDEL syndrome, a recessive disorder of dystonia and deafness with Leigh-like syndrome, impaired oxidative phosphorylation and 3-methylglutaconic aciduria. We localized SERAC1 at the interface between the mitochondria and the endoplasmic reticulum in the mitochondria-associated membrane fraction that is essential for phospholipid exchange. A phospholipid analysis in patient fibroblasts showed elevated concentrations of phosphatidylglycerol-34:1 (where the species nomenclature denotes the number of carbon atoms in the two acyl chains:number of double bonds in the two acyl groups) and decreased concentrations of phosphatidylglycerol-36:1 species, resulting in an altered cardiolipin subspecies composition. We also detected low concentrations of bis(monoacyl-glycerol)-phosphate, leading to the accumulation of free cholesterol, as shown by abnormal filipin staining. Complementation of patient fibroblasts with wild-type human SERAC1 by lentiviral infection led to a decrease and partial normalization of the mean ratio of phosphatidylglycerol-34:1 to phosphatidylglycerol-36:1. Our data identify SERAC1 as a key player in the phosphatidylglycerol remodeling that is essential for both mitochondrial function and intracellular cholesterol trafficking.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Amino Acid Sequence
  • Carboxylic Ester Hydrolases / genetics*
  • Carboxylic Ester Hydrolases / metabolism
  • Cardiolipins / genetics
  • Cardiolipins / metabolism
  • Cell Line, Transformed
  • Cell Line, Tumor
  • Cholesterol / genetics
  • Cholesterol / metabolism*
  • Deafness / genetics*
  • Deafness / metabolism
  • Dystonia / genetics*
  • Dystonia / metabolism
  • Exome
  • Fibroblasts / metabolism
  • HEK293 Cells
  • HeLa Cells
  • Humans
  • Mitochondria / genetics*
  • Mitochondria / metabolism
  • Molecular Sequence Data
  • Mutation*
  • Oxidative Phosphorylation
  • Phosphatidylglycerols / genetics
  • Phosphatidylglycerols / metabolism
  • Phospholipids / genetics
  • Phospholipids / metabolism*
  • Sequence Alignment

Substances

  • Cardiolipins
  • Phosphatidylglycerols
  • Phospholipids
  • Cholesterol
  • Carboxylic Ester Hydrolases
  • SERAC1 protein, human