Characterization of five partial deletions of the factor VIII gene

Proc Natl Acad Sci U S A. 1987 Jun;84(11):3772-6. doi: 10.1073/pnas.84.11.3772.

Abstract

Hemophilia A is an X-linked disorder of coagulation caused by a deficiency of factor VIII. By using cloned DNA probes, we have characterized the following five different partial deletions of the factor VIII gene from a panel of 83 patients with hemophilia A: (i) a 7-kilobase (kb) deletion that eliminates exon 6; (ii) a 2.5-kb deletion that eliminates 5' sequences of exon 14; (iii) a deletion of at least 7 kb that eliminates exons 24 and 25; (iv) a deletion of at least 16 kb that eliminates exons 23-25; and (v) a 5.5-kb deletion that eliminates exon 22. The first four deletions are associated with severe hemophilia A. By contrast, the last deletion is associated with moderate disease, possibly because of in-frame splicing from moderate disease, possibly because of in-frame splicing from adjacent exons. None of those patients with partial gene deletions had circulating inhibitors to factor VIII. One deletion occurred de novo in a germ cell of the maternal grandmother, while a second deletion occurred in a germ cell of the maternal grandfather. These observations demonstrate that de novo deletions of X-linked genes can occur in either male or female gametes.

Publication types

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

MeSH terms

  • Chromosome Deletion*
  • DNA Restriction Enzymes
  • Exons
  • Factor VIII / genetics*
  • Female
  • Genes*
  • Genetic Carrier Screening
  • Hemophilia A / blood
  • Hemophilia A / genetics*
  • Humans
  • Male
  • Pedigree
  • Polymorphism, Genetic

Substances

  • Factor VIII
  • DNA Restriction Enzymes