19Aug 2023
Gene Therapy in Patients with the Crigler–Najjar Syndrome

Gene Therapy in Patients with the Crigler–Najjar Syndrome

Patients with Crigler-Najjar syndrome lack an enzyme leading to severe jaundice. A study tested a gene therapy using an adeno-associated virus in 5 patients. Two doses were tried, and safety and efficacy were evaluated. No major side effects occurred, but some had elevated liver enzymes. The higher dose decreased bilirubin levels even after stopping phototherapy for 78 weeks. This study suggests the potential benefits of gene therapy for Crigler-Najjar syndrome.

  • #family health

Like

Save

Share