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Gene therapy for Crigler-Najjar syndrome - First patient treated in the European clinical trial
Posted on: 25 December 2018, source: Eurekalerts
Following patient recruitment and preliminary observation period, the first patient was injected by Prof. Labrune at Beclere Hospital in Clamart, France. Genethon, the AFM-Telethon laboratory, is sponsoring this multicenter international phase I/II trial, injecting intravenously an AAV vector that is able to transfer the UGT1A1 gene (coding for the production of bilirubin GT) into liver cells. A total of 17 patients will be treated over the next few months.
Following patient recruitment and preliminary observation period, the first patient was injected by Prof. Labrune at Beclere Hospital in Clamart, France. Genethon, the AFM-Telethon laboratory, is sponsoring this multicenter international phase I/II trial, injecting intravenously an AAV vector that is able to transfer the UGT1A1 gene (coding for the production of bilirubin GT) into liver cells. A total of 17 patients will be treated over the next few months.