Hemophilia gene therapy: novel rAAV vectors and RNA repair strategy

Curr Opin Mol Ther. 2002 Oct;4(5):499-504.

Abstract

Hemophilia results from a deficiency of coagulation Factor VIII or IX and manifests clinically as spontaneous bleeding into the large joints and soft tissue. Current treatment relies on the intravenous infusion of recombinant or purified Factor proteins. Factor infusion is effective, but transient due to the short half-life of Factor proteins. Recent developments in gene transfer technology have led to new strategies using molecular therapeutics as permanent treatment for bleeding disorders. This review describes recent novel molecular strategies for the treatment of the hemophilias.

Publication types

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

MeSH terms

  • Dependovirus / genetics*
  • Genetic Therapy*
  • Genetic Vectors*
  • Hemophilia A / therapy*
  • Humans
  • RNA / genetics*
  • RNA Splicing

Substances

  • RNA