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Front Biosci (Landmark Ed). 2015 Jan 1;20:556-603.

Gene therapy for hemophilia.

Author information

1
University of Florida, Department of Pediatrics, Division of Cellular and Molecular Therapy, Gainesville, FL 32610.

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.

PMID:
25553466
PMCID:
PMC4476626
[Indexed for MEDLINE]
Free PMC Article

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