Homozygous factor V splice site mutation associated with severe factor V deficiency

Blood. 2002 Apr 15;99(8):3063-5. doi: 10.1182/blood.v99.8.3063.

Abstract

We investigated a family whose proband has a severe bleeding disorder and factor V antigenic and functional levels of 8% and less than 1% of control values, respectively. Molecular analysis of the factor V gene revealed a novel homozygous mutation in the last nucleotide of exon 10. 1701G>T causes activation of a cryptic exonic splice site in exon 10, which encodes part of the factor V heavy chain (A2 domain). This leads to the deletion of 35 nucleotides and results in a frameshift with a premature stop codon at amino acid position 498. The G1701 and corresponding Gln509 are conserved in murine, bovine, and porcine factor V and in human factor VIII. Few factor V deficiency mutations have been identified as yet. Several are present in the heterozygous form in combination with factor V Leiden (Arg506Gln). This is the first reported homozygous splice site mutation in a patient with factor V deficiency.

Publication types

  • Case Reports

MeSH terms

  • Blood Transfusion
  • Exons
  • Factor V / genetics*
  • Factor V Deficiency / complications
  • Factor V Deficiency / genetics*
  • Factor V Deficiency / therapy
  • Family Health
  • Female
  • Frameshift Mutation
  • Hemorrhage / etiology
  • Hemorrhage / genetics
  • Hemorrhage / therapy
  • Homozygote
  • Humans
  • Male
  • Mutation*
  • Pedigree
  • Point Mutation
  • RNA Splice Sites / genetics*
  • Sequence Deletion

Substances

  • RNA Splice Sites
  • Factor V