Whole-Exome Sequencing Identified a Novel Compound Heterozygous Genotype in ASL in a Chinese Han Patient with Argininosuccinate Lyase Deficiency

Biomed Res Int. 2019 Apr 30:2019:3530198. doi: 10.1155/2019/3530198. eCollection 2019.

Abstract

Pathogenic variants in the argininosuccinate lyase (ASL) gene have been shown to cause argininosuccinate lyase deficiency (ASLD); therefore, sequencing analysis offers advantages for prenatal testing and counseling in families afflicted with this condition. Here, we performed a genetic analysis of an ASLD patient and his family with an aim to offer available information for clinical diagnosis. The research subjects were a 23-month-old patient with a high plasma level of citrulline and his unaffected parents. Whole-exome sequencing identified potential related ASL gene mutations in this trio. Enzymatic activity was detected spectrophotometrically by a coupled assay using arginase and measuring urea production. We identified a novel nonsynonymous mutation (c.206A>G, p.Lys69Arg) and a stop mutation (c.637C>T, p.Arg213∗) in ASL in a Chinese Han patient with ASLD. The enzymatic activity of a p.Lys69Arg ASL construct in human embryonic kidney 293T cells was significantly reduced compared to that of the wild-type construct, and no significant activity was observed for the p.Arg213∗ construct. Compound heterozygous p.Lys69Arg and p.Arg213∗ mutations that resulted in reduced ASL enzyme activity were found in a patient with ASLD. This finding expands the clinical spectrum of ASL pathogenic variants.

Publication types

  • Case Reports

MeSH terms

  • Amino Acid Substitution
  • Argininosuccinate Lyase* / genetics
  • Argininosuccinate Lyase* / metabolism
  • Argininosuccinic Aciduria* / enzymology
  • Argininosuccinic Aciduria* / genetics
  • Citrulline / blood
  • Citrulline / genetics
  • Exome Sequencing*
  • HEK293 Cells
  • Heterozygote*
  • Humans
  • Infant
  • Male
  • Mutation, Missense*

Substances

  • Citrulline
  • Argininosuccinate Lyase