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Showing 1 to 2 of 2 for “"F8 gene"”.
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Improving the Safety and Efficacy of AAV Gene Therapy for Hemophilia A
Gene therapy for hemophilia A (HA) remains an unrealized therapeutic goal due to outstanding questions regarding safety and long-term efficacy after adeno-associated viral (AAV) vector delivery of the F8 gene to hepatocytes. To address questions of safety, we treated privately owned dogs with a …
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COMPUTATIONAL APPROACHES IN STUDYING INHIBITOR DEVELOPMENT IN HAEMOPHILIA A PATIENTS
… legato al cromosoma X, causato da mutazioni nel gene F8, che determinano una carenza o un malfunzionamento del fattore VIII (FVIII) della coagulazione. Una delle principali complicazioni cliniche nei casi gravi è lo sviluppo di alloanticorpi neutralizzanti, noti come inibitori, che compromettono …