CORDIS Project
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CureCN is focused on developing a gene therapy for Crigler-Najjar syndrome, a rare genetic disorder. The project aims to use a viral vector to deliver the UGT1A1 gene to the liver, potentially offering a curative treatment for affected patients.
Crigler-Najjar syndrome (CN) is a rare recessive disorder caused by mutations in the uridine diphosphate glucuronosyltransferase 1A1(UGT1A1) gene. CN is a life-threatening disease with no cure which constitutes a severe burden for the patients, their families, and the society.
CureCN has the objective of developing a curative gene therapy for CN syndrome based on liver gene transfer with and adeno-associated virus (AAV) vector expressing the UGT1A1 transgene.
Additional goals of CureCN are to de…
ASSOCIATION GENETHON
Partner organizations (coordinator is shown above), with normalized type and CORDIS activity type. Guests see up to 4 partners.
ASSISTANCE PUBLIQUE HOPITAUX DE PARIS
France, Paris
Type: Research institute
Activity type: Research Organisations
SME: No
Netherlands, Amsterdam
Type: Research institute
Activity type: Research Organisations
SME: No
Germany, Hannover
Type: University / higher education
Activity type: Higher or Secondary Education Establishments
SME: No
Italy, BERGAMO
Type: Public body
Activity type: Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments)
SME: No
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