The clip is available on the CureCN project website (https://curecn.eu/videos) and on YouTube (https://youtu.be/nk483P0cpKo). Since the project launch in 2018, the 11 international partners from academia, hospitals, healthcare companies and patient organisations are collaborating to find a curative treatment for the CN syndrome. In order to achieve this goal, the project conducts a clinical trial to prove the safety and efficacy of an innovative gene therapy based on liver gene transfer with an adeno-associated virus (AAV). As a patient-driven initiative, the CureCN consortium comprises all active Crigler-Najjar patient organisations in Europe. They provide a direct link to patients and their families enabling a vivid exchange of information to ensure that those affected are directly involved in the research process. During the first period of the project, the CureCN partners have taken significant initial steps towards the realisation of the project’s objectives. Following recruitment and a preliminary observation period, the first participants have been enrolled in the clinical trial which marks an important milestone not only for the patients and their families, but also for doctors and researchers working together in the CureCN consortium.
gene therapy, genetic disease, rare disease, liver disease, AAV, Horizon 2020, crigler-najjar, CN