Improved intravenous lentiviral gene therapy based on endothelial-specific promoter-driven factor VIII expression for hemophilia A

• Päätekijät: Jie Gong (Tekijä), Rui Yang (Tekijä), Min Zhou (Tekijä), Lung-Ji Chang (Tekijä)
• Aineistotyyppi: Kirja
• Julkaistu: BMC, 2023-06-01T00:00:00Z.
• Aiheet: article
• Linkit: Connect to this object online.

Abstract Background Hemophilia A (HA) is an X-linked monogenic disorder caused by deficiency of the factor VIII (FVIII) gene in the intrinsic coagulation cascade. The current protein replacement therapy (PRT) of HA has many limitations including short term effectiveness, high cost, and life-time tre...