Gene Therapy in Patients with the Crigler-Najjar Syndrome.
Lorenzo D'AntigaUlrich BeuersGiuseppe RonzittiNicola Brunetti-PierriUlrich BaumannAngelo Di GiorgioSem AronsonAurelie HubertRoberta RomanoNorman JungePiter BosmaGiulia BortolussiAndrés Fernando MuroRavaka F SoumoudrongaPhilippe VeronFanny CollaudNathalie Knuchel-LegendrePhilippe LabruneFederico MingozziPublished in: The New England journal of medicine (2023)
No serious adverse events were reported in patients treated with the gene-therapy vector GNT0003 in this small study. Patients who received the higher dose had a decrease in bilirubin levels and were not receiving phototherapy at least 78 weeks after vector administration. (Funded by Genethon and others; ClinicalTrials.gov number, NCT03466463.).
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