Scientists at the Mayo Clinic are testing a new approach to correct metabolic liver disorders without a whole-organ transplant. Their findings (“Curative Ex Vivo Liver-Directed Gene Therapy in a Pig Model of Hereditary Tyrosinemia Type 1”) appear in Science Translational Medicine . ❖ Medicilon’s Endocrine & Metabolic Disease Models Services In the field of metabolic diseases, we are proud of our portfolio of stable and effective animal models, especially those for Non-alcoholic Fatty Liver Disease (NAFLD). Though popular recently, they still lack effective drug treatment. The medical research study tested gene therapy in pigs suffering from hereditary tyrosinemia type 1 (HT1), a metabolic disorder caused by an enzyme deficiency. The common treatment for this disease is a drug regimen, but it is ineffective in many patients...
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