Factor IX Fukuoka. Substitution of ASN92 by His in the second epidermal growth factor-like domain results in defective interaction with factors VIIa/X

J Biol Chem. 1993 Nov 15;268(32):24041-6.

Abstract

Hemophilia B Fukuoka, a moderately severe bleeding disorder, is a naturally occurring mutant of factor IX. Plasma from our patient had 3% clotting activity even though 64% of factor IX antigen was present. The purified mutant protein was cleaved normally by factor Xla, factor VIIa-tissue factor complex, or RVV-X (factor X-activating enzyme from Russell's viper venom), yielding a two-chain factor IXa. Amino acid composition and sequence analyses of one of the lysyl endopeptidase peptides derived from factor IX Fukuoka revealed that Asn92 in the second epidermal growth factor (EGF)-like domain had been replaced by His. The active site of the factor IXa Fukuoka was normally competent for the incorporation of p-aminobenzamidine and for the hydrolysis of a synthetic substrate, N alpha-benzyloxycarbonyl-L-arginine p-nitrobenzyl ester. Factor Xa formation by factor IXa Fukuoka was only 8% of the normal factor IXa, even in the presence of polylysine, and only 0.2% of the normal in the system containing phospholipids, Ca2+, and factor VIIIa, thereby indicating a functional defect in interaction of the mutant with factors VIIIa/X. Furthermore, catalytic efficiency (kcat/Km) of factor IXa Fukuoka toward factor X in the presence of Ca2+, phospholipids, and factor VIIIa was only 2.3% of the normal factor IXa. These results suggest that an Asn-to-His substitution at position 92 in the second EGF-like domain of factor IX Fukuoka would have an untoward effect on the specific conformational state of factor IX for binding with factors VIIIa/X.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Asparagine / genetics
  • Benzamidines / metabolism
  • Blood Coagulation Factors / metabolism
  • Epidermal Growth Factor / genetics
  • Esterases / metabolism
  • Factor IX / genetics*
  • Factor IX / metabolism
  • Factor VIIa / metabolism*
  • Factor X / metabolism*
  • Hemophilia B / genetics*
  • Hemophilia B / metabolism
  • Histidine / genetics
  • Humans
  • Hydrolysis
  • Kinetics
  • Male
  • Mutation
  • Peptide Mapping

Substances

  • Benzamidines
  • Blood Coagulation Factors
  • Histidine
  • Epidermal Growth Factor
  • 4-aminobenzamidine
  • Asparagine
  • Factor IX
  • Factor X
  • Esterases
  • Factor VIIa