Trial demonstrates efficacy of gene therapy in patients suffering from Crigler-Najjar syndrome, a rare liver disease

Crigler-Najjar syndrome is characterized by hyperbilirubinemia. If it is not treated quickly, the build-up of bilirubin, due to a deficiency in the enzyme UGT1A1 which is responsible for metabolizing it into a substance that can be eliminated by the body, can cause significant neurological damage and become fatal. At the current time, the only treatment is phototherapy of up to 12 hours per day or a liver transplant.