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This European website, initiated and developed by CSL Behring, has two separate sections with the aim to provide information on haemophilia for an international audience, either to European healthcare professionals or to the general public.*
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- Doshi BS, Arruda VR. Gene therapy for hemophilia: what does the future hold? Ther Adv Hematol. 2018;9(9):273-293.
- Nathwani AC. Gene therapy for hemophilia. Hematology Am Soc Hematol Educ Program. 2019;2019(1):1-8.
- Heinz S, Braspenning J. Measurement of blood coagulation factor synthesis in cultures of human hepatocytes. Methods Mol Biol. 2015;1250:309-16.
- Naso MF, Tomkowicz B, Perry WL III, Strohl WR. Adeno-associated virus AAV as a vector for gene therapy. BioDrugs. 2017;31:317-334.
- Nathwani AC, Reiss UM, Tuddenham EGD, et al. Long-term safety and efficacy of factor IX gene therapy in hemophilia B. N Engl J Med. 2014;371:1994-2004.
- Wang D, Tai PW, Gao G. Adeno-associated virus vector as a platform for gene therapy delivery. Nat Rev Drug Discov. 2019;18(5):358-378.
- Perrin GQ, Herzog RW, Markusic DM. Update on clinical gene therapy for hemophilia. Blood. 2019;133(5):407-414.
- Arruda VR, Doshi BS. Gene therapy for hemophilia: facts and quandaries in the 21st century. Med J Hematol Infect Dis. 2020;12(1):e2020069.
- Arruda V, Stedman H, Jian H, et al. Correction of hemophilia B phenotype by novel method of regional intravenous delivery of AAV vector to skeletal muscle of hemophilia B dogs. Mol Ther. 2005;11:S233.
- Nathwani AC, Tuddenham EGD, Rangarajan S, et al. Adenovirus-associated virus vector-mediated gene transfer in hemophilia B. New Engl J Med. 2011;365(25):2357-2365.
- Nathwani AC, Reiss U, Tuddenham EGD, at al. Adeno-associated mediated gene transfer for hemophilia B: 8 year follow up and impact of removing "empty viral particles" on safety and efficacy of gene transfer. Blood 2018;132(Suppl 1):491.
- High KA, Roncarolo MG. Gene therapy. N Engl J Med. 2019;381(5):455-464.
- Food and Drug Administration (FDA) Cellular, Tissue, and Gene Therapies Advisory Committee Meeting #70. Toxicity risks of adeno-associated virus (AAV) vectors for gene therapy (GT). Published September 2021. https://www.fda.gov/media/151969/download Accessed July 14, 2023.