PC (protein C) is a plasma anticoagulant encoded by PROC; mutation in both PROC alleles results in neonatal purpura fulminans—a fatal systemic thrombotic disorder. In the present study, we aimed to ...

Adeno-associated viruses (AAVs) are promising delivery tools for gene therapy and are used in many clinical studies. However, less than ten AAV-based therapies have been approved to date. To mitigate ...