The overall vision of the European research project CureCN is to provide a curative gene therapy for the ultra-rare Crigler-Najjar syndrome (CN). CN is a life-threatening, so far incurable liver disease that affects about one in a million individuals at birth.
The CureCN consortium joins forces to prove the safety and efficacy of the gene therapy in a clinical trial and to make the treatment available to patients. The project is funded by Horizon 2020 with a total budget of € 6.25 million.
In 2018, the first patient affected by the Crigler-Najjar syndrome was treated in the context of the clinical trial promoted by CureCN.
The ICGEB Mouse Molecular Genetics group, external collaborator to the CureCN consortium (subcontractor of the coordinator GENETHON), has been instrumental in this achievement, thanks to the AAV-gene therapy experiments conducted in the murine model of the disease, developed by the same laboratory.
CureCN project website https://curecn.eu
Duration: 2018-2022
Donor/funding programme:
GENETHON Subcontract of a H2020 financed project
Partners:
Andrés Muro Mouse Molecular Genetics, ICGEB Trieste, Italy