Translational Research for genetic diseases

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Translational Research for genetic diseases

The treatment for some genetic disorders can cause a single protein deficiency. In hemophilia A, which is caused by a deficiency of coagulation factor VIII, can be treated by replacing the missing factor. Unfortunately, the most severely affected patients, who completely lack endogenous factor VIII, often develop antibodies against the replacement protein because their bodies recognize it as a foreign substance. To overcome this problem, now a method is developed for transplacental exposure of fetuses to the missing antigen, thus ensuring immune tolerance for protein therapy that will be delivered after birth.

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