Factor IX-Padua: gene therapy and hemophilia
Factor IX-Padua: gene therapy and hemophilia
Hemophilia is a Mendelian disease (which is inherited by the mutation of a single gene) on the X chromosome, the disease is transmitted as a sex-linked character, occurs mainly in males. It is a recessive genetic disease that prevents the proper coagulation of blood. As a result, the wounds of those affected bleed for a longer period of time, leading to hemorrhages both externally and internally.
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