A truncating mutation in B3GNT1 causes severe Walker-Warburg syndrome

Neurogenetics. 2013 Nov;14(3-4):243-5. doi: 10.1007/s10048-013-0367-8. Epub 2013 Jul 23.

Abstract

Walker-Warburg syndrome (WWS) is a genetically heterogeneous form of congenital muscular dystrophy with significant brain and ocular involvement. In a multiplex consanguineous family with severe WWS phenotype, autozygome-guided sequencing of previously reported WWS genes was negative. Exome sequencing followed by autozygome filtration revealed a homozygous two-base pair insertion in B3GNT1 (NM_006876.2:c.821_822insTT), leading to premature truncation of the protein (p.Glu274Aspfs*94). Recently, two missense mutations in this gene have been reported as probably causal in a family with WWS. This report describes the first truncating mutation in B3GNT1 and confirms that this gene, which plays a role in αDG glycosylation, is a bona fide disease gene in WWS.

Publication types

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

MeSH terms

  • Brain / pathology
  • Female
  • Humans
  • Infant, Newborn
  • Male
  • Mutation*
  • N-Acetylglucosaminyltransferases / genetics*
  • Walker-Warburg Syndrome / genetics*
  • Walker-Warburg Syndrome / pathology

Substances

  • N-Acetylglucosaminyltransferases
  • B4GAT1 protein, human