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Animal research, Health, Scientific progress

25 - 07 - 2016

Soon an effective long-term treatment against Crigler-Najjar disease ?

Researchers from the INSERM and Pierre and Marie Curie University have discovered how to treat, in the long-term, mice and rats suffering from Crigler-Najjar syndrome, a liver disease that causes a deficit in UGT1A1 enzymes, and therefore, causes bilirubin to accumulate in the blood (a toxic yellow pigment). By injecting a therapeutic vector expressing UGT1A1, they helped restore enzymatic activity in the liver and the correct removal of bilirubin for more than one year after injection.