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在患有克里格勒-纳贾尔综合征的患者中进行基因治疗

Lorenzo D'Antiga1, Ulrich Beuers1, Giuseppe Ronzitti1

  • 1From Department of Pediatric Hepatology, Gastroenterology, and Transplantation, Hospital Papa Giovanni XXIII, Bergamo (L.D., A.D.G.), Scuola Superiore Meridionale, Genomics and Experimental Medicine Program (N.B.-P.), Department of Translational Medicine, University of Naples Federico II, Naples (N.B.-P., R.R.), Telethon Institute of Genetics and Medicine, Pozzuoli (N.B.-P.), and the International Center for Genetic Engineering and Biotechnology, Trieste (G.B., A.F.M.) - all in Italy; Tytgat Institute for Liver and Intestinal Research, Department of Hepatology and Gastroenterology, Amsterdam University Medical Centers, University of Amsterdam, Amsterdam (U. Beuers, S.A., P.B.); Université d'Evry, Université Paris-Saclay, INSERM, Genethon, Integrare Research Unit UMR_S951 (G.R., F.C., F.M.) and Genethon (G.R., R.F.S., P.V., F.C., N.K.-L., F.M.), Evry, Assistance Publique-Hôpitaux de Paris, Université Paris-Saclay, Centre de Référence pour les Maladies Rares, Maladies Héréditaires du Métabolisme Hépatique, Hôpital Antoine Béclère, Clamart (A.H., P.L.), and Université Paris-Saclay and INSERM Unité 1195, Le Kremlin Bicêtre (A.H., P.L.) - all in France; the Division for Pediatric Gastroenterology and Hepatology, Department of Pediatric Kidney, Liver, and Metabolic Diseases, Hannover Medical School, Hannover, Germany (U. Baumann, N.J.); and Spark Therapeutics, Philadelphia (F.M.).

The New England journal of medicine
|August 16, 2023
PubMed
概括

基因疗法为克里格勒-纳贾尔综合征提供了有前途的治疗方法,可显著降低患者的胆红素水平. 较高剂量的UGT1A1基因治疗载体导致持续的胆红素减少,而无需光疗.

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