Immunity Against AAV in Crigler Najjar Patient

Study of the Prevalence of Pre-existing Immunity Against the Adeno-associated Virus (AAV) in Patients With Crigler-Najjar Syndrome

The proposed research protocol aims at addressing these points by pre-screening CN patients for their AAV serology in link with their medical history and current medical status. A first objective is to assess the presence of neutralizing AAV antibodies in the serum of CN patients.

Inclusion Criteria:

• The patient has a Crigler-Najjar syndrome
• The molecular diagnosis has been confirmed by UGT1A1 gene DNA sequencing

Exclusion criteria:

• The patient is liver transplanted
• The patient is suffering from severe associated pathologies precluding further enrolment in a future interventional clinical study