Crigler-Najjar Syndrome

Research Pre-clinical phase Phase I or I/II  

Crigler-Najjar Syndrome is a rare genetic liver disorder resulting in neurological complications caused by the neurotoxicity of unconjugated bilirubin. Symptomatic treatment for the condition involves 10-12 hours of phototherapy every day, and the only possible cure is a liver transplant.

This development project, currently in the preclinical stage, is based on the direct administration into the patient of an AAV (adeno-associated virus) vector carrying a normal copy of the mutated gene.

The project is being conducted by Dr Philippe Labrune (AP-HP, Hospital Antoine Béclère, Clamart) and Dr Piter Bosma (AMC Amsterdam, Tytgat Institute, Amsterdam, Holland), in collaboration with AFM and Genethon.