Fix it in one go: enhanced factor IX gene therapy for hemophilia B

• Autores: David Lillicrap
• Localización: Cell, ISSN 0092-8674, Vol. 171, Nº. 7, 2017, págs. 1478-1480
• Idioma: inglés
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• Resumen

  • A phase 1/2 clinical trial of AAV-mediated gene therapy in patients with hemophilia B using an enhanced specific activity factor IX (FIX) transgene reports sustained levels of FIX levels, leading to the near elimination of bleeding for more than a year and without serious adverse side effects. These results are the best outcome to date for hemophilia gene therapy.