Recent Advances in Gene Therapy for Hemophilia: Projecting the Perspectives
Nikita Chernyi,
Darina Gavrilova,
Mane Saruhanyan
et al.
Abstract:One of the well-known X-linked genetic disorders is hemophilia, which could be hemophilia A as a result of a mutation in the F8 (factor VIII) gene or hemophilia B as a result of a mutation in the F9 (factor IX) gene, leading to insufficient levels of the proteins essential for blood coagulation cascade. In patients with severe hemophilia, factor VIII or factor IX activities in the blood plasma are considerably low, estimated to be less than 1%. This is responsible for spontaneous or post-traumatic bleeding epi… Show more
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