Adeno-Associated Virus Vector-Mediated Liver Gene Therapy for Crigler-Najjar Syndrome
Crigler-Najjar (CN) syndrome is a rare recessive disorder caused by mutations in the uridine diphosphate glucuronosyltransferase 1A1 (UGT1A1) gene for which current treatment options are suboptimal and no permanent cure is available except for liver transplantation. To address the unmet medical need, the CureCN project has the ambitious goal of developing a curative AVV (adeno-associated virus) vector-based gene therapy for CN syndrome. At the heart of the project is an open-label, multicenter clinical trial of AAV8-UGT1A1 gene transfer to prove the safety and efficacy of the therapy in severe CN patients.
- Dr Gérald PerretAssociation GenethonEmail