1 The molecular basis of haemophilia A and B
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Cited by (40)
Update on clinical gene therapy for hemophilia
2019, BloodCitation Excerpt :Hemophilia is an X-linked monogenic coagulation disorder resulting from a deficiency in coagulation factors in the intrinsic coagulation cascade.1,2
Immune deviation by mucosal antigen administration suppresses gene-transfer-induced inhibitor formation to factor IX
2006, BloodCitation Excerpt :In protein or gene replacement therapy for the X-linked bleeding disorders hemophilia A and B (deficiency in coagulation factor VIII or IX, respectively), the risk of production of antibodies that are inhibitory to clotting factor activity is a major concern. In conventional therapy, based on intravenous infusion of plasma-derived or recombinant protein, the incidence of inhibitors is 3% to 4% in hemophilia B and 20% to 30% in hemophilia A.1 Treatment of inhibitor patients is complicated and requires expensive bypass reagents such as protein complex concentrates and activated factor VII. Several protocols have been developed to eliminate inhibitors by frequent intravenous infusion of high doses of the clotting factor protein, often in combination with immunoglobulin infusion and immunosuppressive drug therapy.2
Determination of specific CD4 and CD8 T cell epitopes after AAV2- and AAV8-hF.IX gene therapy
2006, Molecular TherapyCitation Excerpt :In the present studies, we found that hF.IX, as a non-self antigen to mice, when delivered by a viral vector, induced a CTL response. These findings have important implications for human hemophilia B gene therapy in that for some patients, normal F.IX is a new antigen [12,23] and can possibly induce a CTL response. One major impact is the identification of particular class I and class II MHC that are required to bind specific regions of AAV2, AAV8, or hF.IX antigen.
Chimeric Mice Engrafted With Canine Hepatocytes Exhibits Similar AAV Transduction Efficiency to Hemophilia B Dog
2022, Frontiers in PharmacologyHaemophilia, state of the art and new therapeutic opportunities, a regulatory perspective
2021, British Journal of Clinical PharmacologyGenome-editing applications in stem cell engineering and regenerative medicine
2021, Genome Editing in Drug Discovery