gene therapy and adeno-associated virus
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Hemophilia and Gene Therapy–Review
Abstract: 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 …
Published in Research and Reviews : A Journal of Medical Science and Technology · Vol. 12, Issue 3, 2024 · pp. 1–5 Read article