IMR Press / FBL / Volume 20 / Issue 3 / DOI: 10.2741/4324

Frontiers in Bioscience-Landmark (FBL) is published by IMR Press from Volume 26 Issue 5 (2021). Previous articles were published by another publisher on a subscription basis, and they are hosted by IMR Press on imrpress.com as a courtesy and upon agreement with Frontiers in Bioscience.

Open Access Review
Gene therapy for hemophilia
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1 University of Florida, Department of Pediatrics, Division of Cellular and Molecular Therapy, Gainesville, FL 32610, USA
Academic Editor:Liting Song
Front. Biosci. (Landmark Ed) 2015, 20(3), 556–603; https://doi.org/10.2741/4324
Published: 1 January 2015
(This article belongs to the Special Issue Gene therapy of some genetic diseases)
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.

Keywords
Gene therapy
Hemophilia
Factor IX
Factor VIII
AAV
Lentivirus
Review
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