Thus, protein-based therapy will be the standard against which the safety and efficacy of gene transfer will be judged.
Despite life-threatening complications of protein-based clotting factor treatment during the last three decades, including acquired immunodeficiency syndrome and hepatitis C, factor infusion is now considered safe, effective, and compatible with a normal lifespan. One of the unanswered questions in hemophilia is whether gene transfer, if successful, will be a safe and effective alternative to standard clotting factor treatment for hemophilia.
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