Exciting new research project, CureCN aims to cure the ultra-rare Crigler-Najjar Syndrome

The new European research project CureCN focuses on developing a curative gene therapy for the ultra-rare liver disease Crigler-Najjar syndrome (CN) and to make the treatment available to patients. CN is a life-threatening, so far incurable liver disease that affects about one in a million individuals at birth. Caused by the deficiency of a liver-specific substance (uridine diphosphate Glucuronosyltransferase 1A1), toxic unconjugated bilirubin is accumulated in serum and body tissue leading to irreversible neurological damage in the brain. Currently, there is no curing treatment available apart from liver transplantation. Treatment with phototherapy reduces the symptoms, but is very debilitating and loses its efficacy over time.

Eleven partners from six European countries of CureCN consortium join forces to prove the safety and efficacy of the gene therapy in a clinical trial and to make the treatment available to patients. “Our goal is to provide a healing treatment for very young patients, as well as for older patients with a possible pre-existing immunity to AAV that has to be eradicated in the gene therapy process,” said Dr Federico Mingozzi, CureCN coordinator and Head of the Immunology and Liver Gene Therapy team at the laboratory Genethon. “If validated in our clinical trial, AAV-mediated gene therapy could not just cure CN but also heal other inherited liver diseases. Our results could thus change the entire field of in vivo gene therapy.”

The Leicester team represented in the CureCN project by Prof Sergey Piletsky and Dr Elena Piletska from the Department of Chemistry contributes to the project with their expertise in design and preparation of nano-sized molecularly imprinted polymers (nanoMIPs) which will be used as synthetic recognition elements in a novel, polymer-based anti-AAV Ig-specific plasmapheresis resin to selectively remove NAbs from the bloodstream of seropositive subjects. In the frame of the CureCN project Leicester team will supervise and train two PhD students who will benefit from their participation in the cutting-edge multi-disciplinary and multi-national project.

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Department of Chemistry
University of Leicester
Leicester, LE1 7RH, UK

Email: chemistry@le.ac.uk

Tel: [+44] (0)116 252 2100

Fax: [+44] (0)116 252 3789

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