GENE THERAPY FOR COAGULATION DISORDERS: CURRENT ADVANCES AND FUTURE PERSPECTIVES
Keywords:
gene therapy, hemophilia A, hemophilia B, von Willebrand disease, coagulation disorders, AAV vectors, bleeding disorders, factor VIII, factor IXAbstract
In this scientific article, inherited bleeding disorders are examined as a significant global health burden affecting millions of individuals worldwide. Recent advances in gene therapy have revolutionized the treatment landscape for coagulation disorders, particularly hemophilia A and B, with emerging applications for other bleeding conditions, including von Willebrand disease and rare factor deficiencies. This study analyzes the current state of gene therapy for coagulation disorders, focusing on molecular mechanisms, clinical trial outcomes, regulatory approvals, therapeutic challenges, and future perspectives. Evidence from multiple clinical trials demonstrates that adeno-associated virus (AAV)-based gene therapy can achieve sustained increases in clotting factor levels, reducing or eliminating the need for prophylactic factor replacement therapy. However, substantial challenges remain, including immune responses, durability of expression, accessibility, and cost considerations. This article synthesizes current knowledge from clinical trials, regulatory documents, and expert guidelines to provide a comprehensive overview of this transformative therapeutic modality.
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