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mRNA therapy corrects defective glutathione metabolism and restores ureagenesis in preclinical argininosuccinic aciduria.
Gurung S, Timmermand OV, Perocheau D, Gil-Martinez AL, Minnion M, Touramanidou L, Fang S, Messina M, Khalil Y, Spiewak J, Barber AR, Edwards RS, Pinto PL, Finn PF, Cavedon A, Siddiqui S, Rice L, Martini PGV, Ridout D, Heywood W, Hargreaves I, Heales S, Mills PB, Waddington SN, Gissen P, Eaton S, Ryten M, Feelisch M, Frassetto A, Witney TH, Baruteau J. Gurung S, et al. Among authors: eaton s. Sci Transl Med. 2024 Jan 10;16(729):eadh1334. doi: 10.1126/scitranslmed.adh1334. Epub 2024 Jan 10. Sci Transl Med. 2024. PMID: 38198573 Free PMC article.
In Utero Gene Therapy (IUGT) Using GLOBE Lentiviral Vector Phenotypically Corrects the Heterozygous Humanised Mouse Model and Its Progress Can Be Monitored Using MRI Techniques.
Shangaris P, Loukogeorgakis SP, Subramaniam S, Flouri C, Jackson LH, Wang W, Blundell MP, Liu S, Eaton S, Bakhamis N, Ramachandra DL, Maghsoudlou P, Urbani L, Waddington SN, Eddaoudi A, Archer J, Antoniou MN, Stuckey DJ, Schmidt M, Thrasher AJ, Ryan TM, De Coppi P, David AL. Shangaris P, et al. Among authors: eaton s. Sci Rep. 2019 Aug 12;9(1):11592. doi: 10.1038/s41598-019-48078-4. Sci Rep. 2019. PMID: 31406195 Free PMC article.
Publisher Correction: In Utero Gene Therapy (IUGT) Using GLOBE Lentiviral Vector Phenotypically Corrects the Heterozygous Humanised Mouse Model and Its Progress Can Be Monitored Using MRI Techniques.
Shangaris P, Loukogeorgakis SP, Subramaniam S, Flouri C, Jackson LH, Wang W, Blundell MP, Liu S, Eaton S, Bakhamis N, Ramachandra DL, Maghsoudlou P, Urbani L, Waddington SN, Eddaoudi A, Archer J, Antoniou MN, Stuckey DJ, Schmidt M, Thrasher AJ, Ryan TM, De Coppi P, David AL. Shangaris P, et al. Among authors: eaton s. Sci Rep. 2019 Dec 24;9(1):20214. doi: 10.1038/s41598-019-55754-y. Sci Rep. 2019. PMID: 31874968 Free PMC article.
Beclin-1-mediated activation of autophagy improves proximal and distal urea cycle disorders.
Soria LR, Gurung S, De Sabbata G, Perocheau DP, De Angelis A, Bruno G, Polishchuk E, Paris D, Cuomo P, Motta A, Orford M, Khalil Y, Eaton S, Mills PB, Waddington SN, Settembre C, Muro AF, Baruteau J, Brunetti-Pierri N. Soria LR, et al. Among authors: eaton s. EMBO Mol Med. 2021 Feb 5;13(2):e13158. doi: 10.15252/emmm.202013158. Epub 2020 Dec 28. EMBO Mol Med. 2021. PMID: 33369168 Free PMC article.
1,187 results