Gene therapy for hemophilia B: host immunosuppression prolongs the therapeutic effect of adenovirus-mediated factor IX expression

Hum Gene Ther. 1995 Aug;6(8):1039-44. doi: 10.1089/hum.1995.6.8-1039.

Abstract

Hemophilia B is caused by a deficiency of blood clotting factor IX (FIX). Previous studies have shown that the delivery of a recombinant adenoviral vector expressing canine FIX (cFIX) resulted in a complete correction of hemophilia B in FIX-deficient dogs, but that cFIX expression decreased to only about 1-2% of normal levels 3 weeks after treatment. In the present study, therapeutic levels of cFIX expression capable of producing a partial correction of hemophilia B were maintained for at least 6 months after the coadministration of the cFIX-expressing adenovirus and the immunosuppressive agent cyclosporin A (CsA). These findings support a recent report (Yang et al., 1994) that host T-cell-mediated immunity against virally transduced cells is a major contributing factor to the transient nature of adenovirus-mediated gene expression in immunocompetent animals. Although a second administration of the cFIX-expressing adenovirus 6 months after the first infusion had only a minimal effect on plasma FIX levels in a dog that had been continuously treated with CsA, the prolonged expression of the transgene indicates that immunosuppression may be applicable in attaining long-term treatment of clinically relevant disorders.

Publication types

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

MeSH terms

  • Adenoviridae / genetics*
  • Adenoviridae / immunology
  • Animals
  • Antibodies, Viral / blood
  • Blood Coagulation
  • Cyclosporine / pharmacology
  • Dogs
  • Factor IX / biosynthesis
  • Factor IX / genetics*
  • Genetic Therapy / methods*
  • Genetic Vectors / genetics
  • Hemophilia B / blood
  • Hemophilia B / therapy*
  • Immunosuppression Therapy*
  • Immunosuppressive Agents / pharmacology
  • Neutralization Tests

Substances

  • Antibodies, Viral
  • Immunosuppressive Agents
  • Cyclosporine
  • Factor IX