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 ...
Aug 17, 2023
0
43
