Mutations in the X-linked coagulation factor VIII (F8) gene lead to haemophilia A of different grades of severity in humans. Approximately half the severe cases are due to intron-22 inversions. For ...
An open-label, phase 1 trial was conducted in six patients with severe hemophilia A. Dermal fibroblasts obtained from each patient by skin biopsy were grown in culture and transfected with a plasmid ...
Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is an adeno-associated virus 5 (AAV5)–based gene-therapy vector containing a coagulation factor VIII complementary DNA driven by a liver-selective promoter.
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