Characterization of molecular defects of Fitzgerald trait and another novel high-molecular-weight kininogen-deficient patient: insights into structural requirements for kininogen expression

Blood. 2003 Jun 1;101(11):4430-6. doi: 10.1182/blood-2002-11-3329. Epub 2003 Feb 6.

Abstract

A 6-year-old male with vertebral-basilar artery thrombosis was recognized to have high-molecular-weight kininogen (HK) deficiency. The propositus had no HK procoagulant activity and antigen (< 1%). Using monoclonal antibodies (Mabs) to kininogen domain 3, the propositus, family members, and Fitzgerald plasma were determined to have detectable low-molecular-weight kininogen. Mabs to HK domains 5 and 6 do not detect HK antigen in the propositus' plasma. The propositus has a single base pair (bp) deletion in cDNA position 1492 of exon 10 affecting amino acid 480 of the mature protein and resulting in a frameshift and a premature stop codon at position 1597 (amino acid 532). Unexpectedly, Mabs to the heavy chain and domain 5 of HK detect a 92-kDa form of HK in Fitzgerald plasma, the first HK-deficient plasma. The 92-kDa Fitzgerald HK has amino acid residues through 502, corresponding to domains 1 through 5, but lacks epitopes of domain 6 (positions 543 to 595). Fitzgerald DNA has a normal exon 10, but a 17-bp mutation in intron 9. These combined results indicate that mutations in the kininogen gene may differentially affect biosynthesis, processing, and/or secretion of HK.

Publication types

  • Case Reports
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Child
  • Codon, Nonsense
  • Exons
  • Family Health
  • Frameshift Mutation
  • Humans
  • Immunoblotting
  • Introns
  • Kininogen, High-Molecular-Weight / biosynthesis
  • Kininogen, High-Molecular-Weight / deficiency*
  • Kininogen, High-Molecular-Weight / genetics
  • Kininogens / biosynthesis
  • Kininogens / genetics
  • Kininogens / metabolism
  • Male
  • Protein Structure, Tertiary
  • Thrombosis / blood
  • Thrombosis / genetics

Substances

  • Codon, Nonsense
  • Kininogen, High-Molecular-Weight
  • Kininogens