Washington. Metabolic defects, caused by various pathologies, can be corrected by administering proteins with enzymatic activity that can replace a deficient function in the body. All drugs used in “enzyme replacement therapy” in humans are based on single enzymes. However, in order to perform complex functions, more enzymes that work co-ordinately in a metabolic pathway may be needed. A research study from the University of Parma funded by the Telethon Foundation is now paving the way for a therapy that can replace a whole metabolic pathway in humans. The results of this study which has so far been able to demonstrate the full in vitro functionality of the three “masked” enzymes, will be published in the July issue of “Pharmaceutical Research” – the official journal of the American Association of Pharmaceutical Scientists. Still awaiting results from in vivo tests on laboratory animals that reproduce the same mutations responsible for human disease (9Colonne)
