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Low-Dose Gene Therapy for Murine PKU Using Episomal Naked DNA Vectors Expressing PAH from Its Endogenous Liver Promoter.
Grisch-Chan HM, Schlegel A, Scherer T, Allegri G, Heidelberger R, Tsikrika P, Schmeer M, Schleef M, Harding CO, Häberle J, Thöny B. Grisch-Chan HM, et al. Among authors: allegri g. Mol Ther Nucleic Acids. 2017 Jun 16;7:339-349. doi: 10.1016/j.omtn.2017.04.013. Epub 2017 Apr 20. Mol Ther Nucleic Acids. 2017. PMID: 28624210 Free PMC article.
Enhancement of hepatic autophagy increases ureagenesis and protects against hyperammonemia.
Soria LR, Allegri G, Melck D, Pastore N, Annunziata P, Paris D, Polishchuk E, Nusco E, Thöny B, Motta A, Häberle J, Ballabio A, Brunetti-Pierri N. Soria LR, et al. Among authors: allegri g. Proc Natl Acad Sci U S A. 2018 Jan 9;115(2):391-396. doi: 10.1073/pnas.1714670115. Epub 2017 Dec 26. Proc Natl Acad Sci U S A. 2018. PMID: 29279371 Free PMC article.
Treatment of a metabolic liver disease by in vivo genome base editing in adult mice.
Villiger L, Grisch-Chan HM, Lindsay H, Ringnalda F, Pogliano CB, Allegri G, Fingerhut R, Häberle J, Matos J, Robinson MD, Thöny B, Schwank G. Villiger L, et al. Among authors: allegri g. Nat Med. 2018 Oct;24(10):1519-1525. doi: 10.1038/s41591-018-0209-1. Epub 2018 Oct 8. Nat Med. 2018. PMID: 30297904 Free article.
Comprehensive characterization of ureagenesis in the spfash mouse, a model of human ornithine transcarbamylase deficiency, reveals age-dependency of ammonia detoxification.
Allegri G, Deplazes S, Rimann N, Causton B, Scherer T, Leff JW, Diez-Fernandez C, Klimovskaia A, Fingerhut R, Krijt J, Kožich V, Nuoffer JM, Grisch-Chan HM, Thöny B, Häberle J. Allegri G, et al. J Inherit Metab Dis. 2019 Nov;42(6):1064-1076. doi: 10.1002/jimd.12068. Epub 2019 Mar 13. J Inherit Metab Dis. 2019. PMID: 30714172 Review.
A liver-humanized mouse model of carbamoyl phosphate synthetase 1-deficiency.
Srinivasan RC, Zabulica M, Hammarstedt C, Wu T, Gramignoli R, Kannisto K, Ellis E, Karadagi A, Fingerhut R, Allegri G, Rüfenacht V, Thöny B, Häberle J, Nuoffer JM, Strom SC. Srinivasan RC, et al. Among authors: allegri g. J Inherit Metab Dis. 2019 Nov;42(6):1054-1063. doi: 10.1002/jimd.12067. Epub 2019 Apr 2. J Inherit Metab Dis. 2019. PMID: 30843237 Free article.
149 results