Periodic Reporting for period 3 - CureCN (Adeno-Associated Virus Vector-Mediated Liver Gene Therapy for Crigler-Najjar Syndrome)

The CureCN project aims to develop a curative gene therapy against the ultra-rare Crigler-Najjar syndrome (CN). CN is a life-threatening liver disease which affects one in a million individuals at birth. It is caused by the deficiency of a liver-specific enzyme (uridine...