haemophilla : monogenic disorder : :_________ : mitochondrial disorder
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Gene therapy can potentially fulfill this need, because (1) hemophilia is an inherited monogenic disease, (2) its symptoms can be improved with a 1 % increase of in vivo coagulation activity, and (3) the patient's phenotype can be improved through a stable supply of therapeutic proteins, such as FVIII and FIX, instead
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