title: Gene therapy for hemophilia.
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.
authors.affiliation: University of Florida, Department of Pediatrics, Division of Cellular and Molecular Therapy, Gainesville, FL 32610.
authors.forename: Geoffrey L
authors.initials: GL
authors.lastname: Rogers
authors.affiliation: University of Florida, Department of Pediatrics, Division of Cellular and Molecular Therapy, Gainesville, FL 32610.
authors.forename: Roland W
authors.initials: RW
authors.lastname: Herzog
mesh_terms: D015316:Genetic Therapy; D005822:Genetic Vectors; D006467:Hemophilia A / Q000628:therapy*; D002836:Hemophilia B / Q000628:therapy*; D006801:Humans