Gene Therapy in Patients with the Crigler–Najjar Syndrome

The Crigler–Najjar syndrome is caused by loss-of-function variants in UGT1A1 and can result in hyperbilirubinemia, jaundice, neurologic injury, and death. In this study, gene therapy was examined in five patients.

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Bibliographic Details
Published inThe New England journal of medicine Vol. 389; no. 7; pp. 620 - 631
Main Authors D’Antiga, Lorenzo, Beuers, Ulrich, Ronzitti, Giuseppe, Brunetti-Pierri, Nicola, Baumann, Ulrich, Di Giorgio, Angelo, Aronson, Sem, Hubert, Aurelie, Romano, Roberta, Junge, Norman, Bosma, Piter, Bortolussi, Giulia, Muro, Andrés F., Soumoudronga, Ravaka F., Veron, Philippe, Collaud, Fanny, Knuchel-Legendre, Nathalie, Labrune, Philippe, Mingozzi, Federico
Format Journal Article
LanguageEnglish
Published Boston Massachusetts Medical Society 17.08.2023
Subjects
Adverse events
Alanine transaminase
Bilirubin
Biopsy
Body weight
Data analysis
Enrollments
Enzymes
Gastroenterology
Gastroenterology General
Gene therapy
Genetics
Genetics General
Genomes
Glucuronosyltransferase
Hyperbilirubinemia
Immune response
Jaundice
Laboratories
Life Sciences
Light therapy
Liver
Liver Disease
Liver transplantation
Neurology
Neurosurgery
Neurosurgery General
Patients
Phototherapy
Software
Transplants & implants
Uridine
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Summary:The Crigler–Najjar syndrome is caused by loss-of-function variants in UGT1A1 and can result in hyperbilirubinemia, jaundice, neurologic injury, and death. In this study, gene therapy was examined in five patients.
Bibliography:ObjectType-Article-1
SourceType-Scholarly Journals-1
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ISSN:0028-4793
1533-4406
DOI:10.1056/NEJMoa2214084