Late-onset ornithine transcarbamylase deficiency caused by a somatic mosaic mutation

Hum Genome Var. 2018 Aug 16:5:22. doi: 10.1038/s41439-018-0022-x. eCollection 2018.

Abstract

An 18-month-old boy was diagnosed with late-onset ornithine transcarbamylase deficiency. Genetic analysis revealed a mosaic frameshift mutation (p.Q279fs) in the OTC gene. Despite the presence of a null mutation, he exhibited a milder phenotype, suggesting that the wild-type allele could rescue the function of OTC. The presence of mosaicism has great effects on the clinical phenotype and recurrence-risk assessment, which should be taken into consideration for genetic counseling.

Publication types

  • Case Reports