A novel stop codon mutation in exon 1 (558C>A) of the UGT1A1 gene in a Thai neonate with Crigler-Najjar syndrome type I

Genet Mol Res. 2015 Jan 23;14(1):419-25. doi: 10.4238/2015.January.23.15.

Abstract

Human uridine 5'-diphosphate-glucuronosyltransferases play a critical role in detoxification by conjugating bilirubin with glucoronic acid. Impaired or reduced enzymatic activity causes a spectrum of clinical disorders such as Crigler-Najjar syndrome type I (CN1), Crigler-Najjar syndrome type II, and Gilbert's syndrome. CN1 is a severe form of unconjugated hyperbilirubinemia caused by homozygous or compound heterozygous mutations in the gene for uridine 5'-diphosphate glucuronosyltransferase 1 family, polypeptide A1 (UGT1A1), resulting in complete loss of enzyme function. Here, we report a novel homozygous mutation of UGT1A1 in a female Thai infant who was diagnosed with CN1, and her parents were found to be heterozygous carriers. The patient was homozygous for the c.558C>A mutation, which resulted in a premature stop codon in exon 1. Her asymptomatic parents were carriers of the nonsense c.558C>A mutation. Our result suggests an important role for homozygous c.558C>A mutations in the UGT1A1 gene in the development of severe unconjugated hyperbilirubinemia.

Publication types

  • Case Reports
  • Research Support, Non-U.S. Gov't

MeSH terms

  • Asian People / genetics*
  • Base Sequence
  • Codon, Terminator / genetics*
  • Crigler-Najjar Syndrome / genetics*
  • Crigler-Najjar Syndrome / physiopathology
  • DNA Mutational Analysis
  • Exons / genetics*
  • Female
  • Glucuronosyltransferase / genetics*
  • Humans
  • Infant
  • Infant, Newborn
  • Liver Function Tests
  • Magnetic Resonance Imaging
  • Molecular Sequence Data
  • Mutation / genetics*

Substances

  • Codon, Terminator
  • UGT1A1 enzyme
  • Glucuronosyltransferase

Supplementary concepts

  • Crigler Najjar syndrome, type 2