Open Access
| Issue |
Med Sci (Paris)
Volume 39, Number 11, Novembre 2023
|
|
|---|---|---|
| Page(s) | 833 - 835 | |
| Section | Nouvelles | |
| DOI | https://doi.org/10.1051/medsci/2023159 | |
| Published online | 29 November 2023 | |
- Nguyen N, Bonzo JA, Chen S, et al. Disruption of the Ugt1 locus in mice resembles human Crigler-Najjar type I disease. J Biol Chem 2008 ; 283 : 7901–7911. [CrossRef] [PubMed] [Google Scholar]
- Bellodi-Privato M, Aubert D, Pichard V, et al. Successful gene therapy of the Gunn rat by in vivo neonatal hepatic gene transfer using murine oncoretroviral vectors. Hepatology 2005 ; 42 : 431–438. [CrossRef] [PubMed] [Google Scholar]
- Nguyen TH, Bellodi-Privato M, Aubert D, et al. Therapeutic lentivirus-mediated neonatal in vivo gene therapy in hyperbilirubinemic Gunn rats. Mol Ther 2005 ; 12 : 852–859. [CrossRef] [PubMed] [Google Scholar]
- Toietta G, Mane VP, Norona WS, et al. Lifelong elimination of hyperbilirubinemia in the Gunn rat with a single injection of helper-dependent adenoviral vector. Proc Natl Acad Sci USA. 2005; 102 : 3930 [CrossRef] [PubMed] [Google Scholar]
- Askari FK, Hitomi Y, Mao M, Wilson JM. Complete correction of hyperbilirubinemia in the Gunn rat model of Crigler-Najjar syndrome type I following transient in vivo adenovirus-mediated expression of human bilirubin UDP-glucuronosyltransferase. Gene Ther 1996 ; 3 : 381–388. [PubMed] [Google Scholar]
- Seppen J, Bakker C, de Jong B, et al. Adeno-associated virus vector serotypes mediate sustained correction of bilirubin UDP glucuronosyltransferase deficiency in rats. Mol Ther 2006 ; 13 : 1085–1092. [CrossRef] [PubMed] [Google Scholar]
- Bortolussi G, Zentilin L, Baj G, et al. Rescue of bilirubin-induced neonatal lethality in a mouse model of Crigler-Najjar syndrome type I by AAV9-mediated gene transfer. FASEB J 2012 ; 26 : 1052–1063. [CrossRef] [PubMed] [Google Scholar]
- D’Antiga L, Beuers U, Ronzitti G, et al. Gene therapy in patients with the Crigler-Najjar syndrome. N Engl J Med 2023; 389 : 620–31. [CrossRef] [PubMed] [Google Scholar]
- Chowdary P, Shapiro S, Makris M, et al. Phase 1–2 trial of AAVS3 gene therapy in patients with hemophilia B. N Engl J Med 2022; 387 : 237–47. [CrossRef] [PubMed] [Google Scholar]
- Shen W, Liu S, Ou L. rAAV immunogenicity, toxicity, and durability in 255 clinical trials: A meta-analysis. Front Immunol 2022; 27 : 1001263. [CrossRef] [PubMed] [Google Scholar]
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