Gene therapy for hemophilia

Front Biosci (Landmark Ed). 2015 Jan 1;20(3):556-603. doi: 10.2741/4324.

Abstract

Hemophilia is an X-linked inherited bleeding disorder consisting of two classifications, hemophilia A and hemophilia B, depending on the underlying mutation. Although the disease is currently treatable with intravenous delivery of replacement recombinant clotting factor, this approach represents a significant cost both monetarily and in terms of quality of life. Gene therapy is an attractive alternative approach to the treatment of hemophilia that would ideally provide life-long correction of clotting activity with a single injection. In this review, we will discuss the multitude of approaches that have been explored for the treatment of both hemophilia A and B, including both in vivo and ex vivo approaches with viral and nonviral delivery vectors.

Publication types

  • Research Support, N.I.H., Extramural
  • Review

MeSH terms

  • Genetic Therapy*
  • Genetic Vectors
  • Hemophilia A / therapy*
  • Hemophilia B / therapy*
  • Humans