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Hemophilia and Gene Therapy–Review

Ashish Rao M, Kusalik Boppana, Nivedita .


The coagulation factor VIII or factor IX genes are affected by the monogenic mutational illness known as hemophilia. Recombinant clotting factors, which are secure and efficient, are the preferred palliative therapy. In order to ensure steady and long-lasting concentrations of the damaged circulating factor, advanced therapies will be curative. Adeno-associated vectors have primarily been used, and the results have been positive in terms of amounts and timings of expression. These treatments, though, are linked to hepatotoxicity and immunogenicity. The focal aim of this review is to provide information regarding the disease pathophysiology and the characteristics of hemophilia A, along with the current treatments.


Hemophilia, FIX transgene; FVIII transgene; gene therapy and adeno-associated virus, diseases.

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