The hemophilias--from royal genes to gene therapy

N Engl J Med. 2001 Jun 7;344(23):1773-9. doi: 10.1056/NEJM200106073442307.
No abstract available

Publication types

  • Review

MeSH terms

  • Disease Transmission, Infectious / prevention & control
  • Factor IX / chemistry
  • Factor IX / genetics
  • Factor IX / therapeutic use*
  • Factor VIII / chemistry
  • Factor VIII / genetics
  • Factor VIII / therapeutic use*
  • Genetic Therapy
  • Hemophilia A / complications
  • Hemophilia A / genetics
  • Hemophilia A / therapy*
  • Humans
  • Mutation
  • Protein Conformation
  • Recombinant Proteins / therapeutic use
  • Virus Diseases / prevention & control
  • Virus Diseases / transmission

Substances

  • Recombinant Proteins
  • Factor VIII
  • Factor IX