WFH State‐of‐the‐art paper 2020: In vivo lentiviral vector gene therapy for haemophilia

Abstract excerpt

Over the last decade, the development of new treatments for haemophilia has progressed at a very rapid pace. Despite all the promising advances in protein products, the prospect offered by gene therapy of a single potentially lifelong treatment remains attractive for people with haemophilia. Transfer to the liver of coagulation factor VIII (FVIII) or factor IX (FIX) transgenes has indeed the potential to stably...

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