Crigler-Najjar disease is a rare genetic disease of the liver leading to neurologic complications due to the neurotoxicity of non-conjugated bilirubin. Symptomatic treatment of the disease consists in daily phototherapy for 10 to 12 hours.
Pre-clinical study for a gene therapy treatment
- Reference laboratory: Genethon
This development project currently at the pre-clinical stage is based on direct administration of an AAV (Adeno-Associated Virus) vector carrying a normal copy of the gene which is mutated in patients. It is being performed in collaboration with Dr. Philippe Labrune (AP-HP, Antoine Béclère Hospital in Clamart) and Dr. Piter Bosma (AMC Amsterdam Academic Medical Center, Tytgat Institute in Amsterdam).