Truncated UDP-glucuronosyltransferase (UGT) from a Crigler-Najjar syndrome type II patient colocalizes with intact UGT in the endoplasmic reticulum

J Hum Genet. 2014 Mar;59(3):158-62. doi: 10.1038/jhg.2013.138. Epub 2014 Jan 9.

Abstract

Mutations in the gene encoding bilirubin UDP-glucuronosyltransferase (UGT1A1) are known to cause Crigler-Najjar syndrome type II (CN-II). We previously encountered a patient with a nonsense mutation (Q331X) on one allele and with no other mutations in the promoter region or other exons, and proposed that CN-II is inherited as a dominant trait due to the formation of a heterologous subunit structure comprised of the altered UGT1A1 gene product (UGT1A1-p.Q331X) and the intact UGT1A1. Here, we investigated the molecular basis of CN-II in this case by expressing UGT1A1-p.Q331X in cells. UGT1A1-p.Q331X overexpressed in Escherichia coli or mammalian cells directly bound or associated with intact UGT1A1 in vitro or in vivo, respectively. Intact UGT1A1 was observed as a dimer using atomic force microscopy. Fluorescent-tagged UGT1A1-p.Q331X and intact UGT1A1 were colocalized in 293T cells, and fluorescence recovery after photobleaching analysis showed that UGT1A1-p.Q331X was retained in the endoplasmic reticulum (ER) without rapid degradation. These findings support the idea that UGT1A1-p.Q331X and UGT1A1 form a dimer and provide an increased mechanistic understanding of CN-II.

MeSH terms

  • Crigler-Najjar Syndrome / enzymology*
  • Crigler-Najjar Syndrome / metabolism*
  • Endoplasmic Reticulum / enzymology*
  • Glucuronosyltransferase / chemistry
  • Glucuronosyltransferase / genetics
  • Green Fluorescent Proteins / metabolism
  • HEK293 Cells
  • Humans
  • Mutant Proteins / metabolism*
  • Photobleaching
  • Protein Binding
  • Protein Transport
  • Subcellular Fractions / metabolism

Substances

  • Mutant Proteins
  • enhanced green fluorescent protein
  • Green Fluorescent Proteins
  • UGT1A1 enzyme
  • Glucuronosyltransferase

Supplementary concepts

  • Crigler Najjar syndrome, type 2