Literaturhinweise

ALDURAZYM [Packungsbeilage].Novato, CA: BioMarin Pharmaceutical Inc; 2013.

All about the Human Genome Project. Website des National Human Genome Research Institute.
https://www.genome.gov/10001772/all-about-the-human/genome-project-hgp.
Aktualisierung 01.10.2015. Zugriff 11.09.2018.

ASH Clinical News. Researchers report first successful gene therapy for hemophilia A. Website der American Society of Hematology. https://www.ashclinicalnews.org/on-location/researchers-report-first-successful-gene-therapy-hemophilia/ .Zugriff 11.09.2018.

Brineura [Packungsbeilage]. Novato, CA: BioMarin Pharmaceutical Inc; 2018.

Current trends update: acquired immunodeficiency syndrome (AIDS) among patients with hemophilia—United States. Website der Centers for Disease Control. https://www.cdc.gov/mmwr/preview/mmwrhtml/00000181.htm. Zugriff 11.09.2018.

Franklin RE, Gosling RG. Molecular configuration in sodium thymonucleate. Nature. 1953;171(4356):740-741.

Gene therapy—a revolution in progress: human genetics and medical research. Website der National Institutes of Health. https://history.nih.gov/exhibits/genetics/sect4.htm. Zugriff 11.09.2018

Genetic timeline. Website des National Human Genome Research Institute. https://www.genome.gov/pages/education/genetictimeline.pdf. Zugriff 11.09.2018.

Giangrande P. The future of hemophilia treatment: longer-acting factor concentrates versus gene therapy. Semin Thromb Hemost. 2016;42(5):513-517.

Gregor Mendel: the father of modern genetics. Website des Office of NIH History. https://history.nih.gov/exhibits/nirenberg/HS1_mendel.htm. Zugriff 11.09.2018.

Hemophilia: from plasma to recombinant factors. Website der American Society of Hematology. http://www.hematology.org/About/History/50-Years/1524.aspx. Zugriff 11.09.2018.

Highlights of transfusion medicine history. Website der AABB. http://www.aabb.org/tm/Pages/highlights.apx. Zugriff 11.09.2018.

History. Website von BioMarin. http://www.biomarin.com/about/history/#1997. Zugriff 11.09.2018.

History of bleeding disorders. Website der National Hemophilia Foundation. https://www.hemophilia.org/Bleeding-Disorders/History-of-Bleeding-Disorders. Zugriff 11.09.2018.

Hoffman M. FVIIa: you’ve come a long way, baby! Blood. 2008;112(8):3002-3003.

KUVAN [Packungsbeilage]. Novato, CA: BioMarin Pharmaceutical Inc; 2016.

Manno CS, et al. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med. 2006;12(3):342-347.

Morfini M, Coppola A, Franchini M, Di Minno G. Clinical use of factor VIII and factor IX concentrates. Blood Transfus. 2013;11(suppl 4):s55-s63.

NAGLAZYM [Packungsbeilage]. Novato, CA: BioMarin Pharmaceutical Inc; 2013.

Ng HJ, Lee LH. Recombinant activated clotting factor VII (rFVIIa) in the treatment of surgical and spontaneous bleeding episodes in hemophilic patients. Vasc Health Risk Manag. 2006;2(4):433-440.

Powell JS. Longer-acting clotting factor concentrates for hemophilia. J Thromb Haemost. 2015;13(suppl 1):S167-S175.

Srivastava A, Brewer AK, Mauser-Bunschoten EP, et al. Guidelines for the management of hemophilia. Haemophilia. 2013;19(1):e1-e47.

Tratschin J-D, West MH, Sandbank T, Carter BJ. A human parvovirus, adeno-associated virus, as a eucaryotic vector: transient expression and encapsidation of the procaryotic gene for chloramphenicol acetyltransferase. Mol Cell Biol. 1984;4(10):2072-2081.

VIMIZIM [Packungsbeilage]. Novato, CA: BioMarin Pharmaceutical Inc; 2014.

Watson JD, Crick FH. Molecular structure of nucleic acids: a structure for deoxyribose nucleic acid. Nature. 1953;171(4356):737-738.

Wood WI, Capon DJ, Simonsen CC, et al. Expression of active human factor VIII from recombinant DNA clones. Nature. 1984;312(5992):330-337.