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Gene Therapy for Hemophilias: Opportunities and Challenges for the Clinical Laboratory

7 Aug 2025

Abstract excerpt

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). More recently, gene therapy (GT) has been developed and licensed for use in patients. CONTENT: In...

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Hemophilia Treatment and Research
RNA Interference and Gene Delivery
Virus-based gene therapy research

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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PMID: 40795181
DOI: 10.1093/clinchem/hvaf085

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