A three generation X-linked family with Kabuki syndrome phenotype and a frameshift mutation in KDM6A

Am J Med Genet A. 2014 May;164A(5):1289-92. doi: 10.1002/ajmg.a.36442. Epub 2014 Mar 24.

Abstract

Kabuki syndrome is a rare malformation syndrome characterized by a typical facial appearance, skeletal anomalies, cardiac malformation, and mild to moderate intellectual disability. In 55-80% of patients with Kabuki syndrome, a mutation in MLL2 is identified. Recently, eight patients with Kabuki syndrome and a mutation in KDM6A were described. In this report, we describe two brothers with a mutation in KDM6A inherited from their mother and maternal grandmother. The two boys have Kabuki-like phenotypes whereas the mother and grandmother present with attenuated phenotypes. This family represents the first instance of hereditary X-linked Kabuki syndrome. We present a short literature review of the patients described with a mutation in KDM6A.

Keywords: KDM6A; Kabuki syndrome; X-linked; seizure.

Publication types

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

MeSH terms

  • Abnormalities, Multiple / diagnosis*
  • Abnormalities, Multiple / genetics*
  • Adult
  • Face / abnormalities*
  • Facies
  • Female
  • Frameshift Mutation*
  • Genes, X-Linked*
  • Hematologic Diseases / diagnosis*
  • Hematologic Diseases / genetics*
  • Histone Demethylases / genetics*
  • Humans
  • Infant, Newborn
  • Male
  • Middle Aged
  • Nuclear Proteins / genetics*
  • Pedigree
  • Phenotype*
  • Pregnancy
  • Prenatal Diagnosis
  • Vestibular Diseases / diagnosis*
  • Vestibular Diseases / genetics*

Substances

  • Nuclear Proteins
  • Histone Demethylases
  • KDM6A protein, human

Supplementary concepts

  • Kabuki syndrome