Gene Therapy for Hemophilias: Opportunities and Challenges for the Clinical Laboratory
Clin Chem. 2025 Aug 7:hvaf085. doi: 10.1093/clinchem/hvaf085. Online ahead of print. ABSTRACT BACKGROUND: Hemophilia A and B are X-linked coagulation disorders characterized by low or dysfunctional Factor VIII (FVIII) or IX (FIX), respectively. Currently, hemophilia A and B are treated by FVIII/FIX replacement therapy with plasma-derived or recombinant products or by FVIII-mimetic agents (e.g., emicizumab). … Read more