Novel HSAN1 mutation in serine palmitoyltransferase resides at a putative phosphorylation site that is involved in regulating substrate specificity

Neuromolecular Med. 2015 Mar;17(1):47-57. doi: 10.1007/s12017-014-8339-1. Epub 2015 Jan 8.

Abstract

1-Deoxysphingolipids (1-deoxySL) are atypical sphingolipids that are formed by the enzyme serine palmitoyltransferase (SPT) due to a promiscuous use of L-alanine over its canonical substrate L-serine. Several mutations in SPT are associated with the hereditary sensory and autonomic neuropathy type I (HSAN1). The current hypothesis is that these mutations induce a permanent shift in the affinity from L-serine toward L-alanine which results in a pathologically increased 1-deoxySL formation in HSAN1 patients. Also, wild-type SPT forms 1-deoxySL under certain conditions, and elevated levels were found in individuals with the metabolic syndrome and diabetes. However, the molecular mechanisms which control the substrate shift of the wild-type enzyme are not understood. Here, we report a novel SPTLC2-S384F variant in two unrelated HSAN1 families. Affected patients showed elevated plasma 1-deoxySL levels and expression of the S384F mutant in HEK293 cells increased 1-deoxySL formation. Previously, S384 has been reported as one of the two (S384 and Y387) putative phosphorylation sites in SPTLC2. The phosphorylation of wild-type SPTLC2 was confirmed by isoelectric focusing. The impact of an S384 phosphorylation on SPT activity was tested by creating mutants mimicking either a constitutively phosphorylated (S384D, S384E) or non-phosphorylated (S384A, Y387F, Y387F+S384A) protein. The S384D but not the S384E variant was associated with increased 1-deoxySL formation. The other mutations had no influence on activity and substrate affinity. In summary, our data show that S384F is a novel mutation in HSAN1 and that the substrate specificity of wild-type SPT might by dynamically regulated by a phosphorylation at this position.

Publication types

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

MeSH terms

  • Aged
  • Amino Acid Sequence
  • Amino Acid Substitution
  • Conserved Sequence
  • Electrophoresis, Gel, Two-Dimensional
  • Female
  • HEK293 Cells
  • Hereditary Sensory and Autonomic Neuropathies / genetics*
  • Heterozygote
  • Homozygote
  • Humans
  • Isoelectric Focusing
  • Male
  • Middle Aged
  • Models, Molecular
  • Molecular Sequence Data
  • Neural Conduction
  • Pedigree
  • Phosphorylation
  • Phosphoserine / metabolism
  • Protein Processing, Post-Translational
  • Recombinant Fusion Proteins / metabolism
  • Sequence Alignment
  • Sequence Homology, Amino Acid
  • Serine C-Palmitoyltransferase / chemistry
  • Serine C-Palmitoyltransferase / genetics*
  • Serine C-Palmitoyltransferase / physiology
  • Species Specificity
  • Sphingolipids / metabolism
  • Substrate Specificity

Substances

  • Recombinant Fusion Proteins
  • Sphingolipids
  • Phosphoserine
  • SPTLC1 protein, human
  • Serine C-Palmitoyltransferase