title: Gene therapy in an era of emerging treatment options for hemophilia B.
abstract: Factor IX deficiency (hemophilia B) is less common than factor VIII deficiency (hemophilia A), and innovations in therapy for hemophilia B have generally lagged behind those for hemophilia A. Recently, the first sustained correction of the hemophilia bleeding phenotype by clotting factor gene therapy has been described using recombinant adeno-associated virus (AAV) to deliver factor IX. Despite this success, many individuals with hemophilia B, including children, men with active hepatitis, and individuals who have pre-existing natural immunity to AAV, are not eligible for the current iteration of hemophilia B gene therapy. In addition, recent advances in recombinant factor IX protein engineering have led some hemophilia treaters to reconsider the urgency of genetic cure. Current clinical and preclinical approaches to advancing AAV-based and alternative approaches to factor IX gene therapy are considered in the context of current demographics and treatment of the hemophilia B population.
authors.affiliation: Gene Therapy Center, University of North Carolina, Chapel Hill, NC, USA.|Department of Pediatrics, University of North Carolina, Chapel Hill, NC, USA.|Harold R. Roberts Comprehensive Hemophilia Diagnostic and Treatment Center, University of North Carolina, Chapel Hill, NC, USA.
authors.forename: P E
authors.initials: PE
authors.lastname: Monahan
mesh_terms: D000818:Animals; D000229:Dependovirus / Q000235:genetics; D005164:Factor IX / Q000096:biosynthesis / Q000235:genetics / Q000627:therapeutic use; D018014:Gene Transfer Techniques; D020022:Genetic Predisposition to Disease; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors; D002836:Hemophilia B / Q000097:blood / Q000175:diagnosis / Q000235:genetics / Q000628:therapy*; D006490:Hemostatics / Q000627:therapeutic use; D006801:Humans; D016086:Lentivirus / Q000235:genetics; D008297:Male; D011994:Recombinant Proteins / Q000627:therapeutic use; D016896:Treatment Outcome
chemical_list: D006490:Hemostatics; D011994:Recombinant Proteins; D005164:Factor IX
keywords: clinical trial; dependovirus; factor IX; genetic therapy; hemophilia B