ESGCT 2021 | The unmet medical need for Crigler-Najjar syndrome

Lorenzo D’Antiga, MD, Hospital Papa Giovanni XXIII, Bergamo, Italy, describes Crigler-Najjar syndrome (CN), an inherited liver metabolism disorder cause by a lack of hepatic uridine diphosphoglucuronate glucuronosyltransferase 1A1 (UGT1A1), which results in severe unconjugated hyperbilirubinemia that can cause irreversible neurological injury and death. The current standard-of-care is daily phototherapy, however this treatment becomes less effective with increasing age, making liver transplantation ultimately necessary. This has prompted the investigation of gene therapy approaches with the aim of finding novel, less invasive curative strategies for CN. This interview took place at the European Society of Gene & Cell Therapy (ESGCT) Virtual Congress 2021.