Novel mutation of OCRL1 in Lowe syndrome

Indian J Pediatr. 2015 Jan;82(1):89-92. doi: 10.1007/s12098-014-1581-6. Epub 2014 Oct 10.

Abstract

Lowe syndrome is a rare, X-linked recessive genetic disease with multi-organ involvement. The pathogenic gene is OCRL1. The authors analyzed the OCRL1 mutation and summarized the clinical features of a Chinese child with Lowe syndrome. The patient is a 3 year 7 mo-old boy. He presented with hypotonia at birth and gradually presented with bilateral congenital cataracts, psychomotor retardation, hypophosphatemic rickets and renal tubular function disorder. Sequence analysis of OCRL1 revealed a novel insertion mutation, c.2367insA (p. Ala813X), in exon 22. This mutation was suspected to cause a premature stop codon of OCRL1 and truncation of the OCRL1 protein. His mother, who carried a heterozygous mutation, had no sign of abnormality.

Publication types

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

MeSH terms

  • Asian People / genetics
  • Cataract* / congenital
  • Cataract* / diagnosis
  • Child, Preschool
  • Codon, Nonsense
  • Familial Hypophosphatemic Rickets / diagnosis*
  • Genetic Predisposition to Disease
  • Humans
  • Intellectual Disability* / diagnosis
  • Intellectual Disability* / etiology
  • Male
  • Mutation
  • Oculocerebrorenal Syndrome* / diagnosis
  • Oculocerebrorenal Syndrome* / genetics
  • Oculocerebrorenal Syndrome* / physiopathology
  • Phosphates / blood
  • Phosphoric Monoester Hydrolases / genetics*
  • Renal Insufficiency* / diagnosis
  • Renal Insufficiency* / etiology

Substances

  • Codon, Nonsense
  • Phosphates
  • Phosphoric Monoester Hydrolases
  • OCRL protein, human