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Year Number of Results
2020 4
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Page 1
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. 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.
Novel therapies for mucopolysaccharidosis type III.
Seker Yilmaz B, Davison J, Jones SA, Baruteau J. Seker Yilmaz B, et al. J Inherit Metab Dis. 2021 Jan;44(1):129-147. doi: 10.1002/jimd.12316. Epub 2020 Sep 28. J Inherit Metab Dis. 2021. PMID: 32944950 Free PMC article. Review.
Clinical applications for exosomes: Are we there yet?
Perocheau D, Touramanidou L, Gurung S, Gissen P, Baruteau J. Perocheau D, et al. Br J Pharmacol. 2021 Jun;178(12):2375-2392. doi: 10.1111/bph.15432. Epub 2021 May 3. Br J Pharmacol. 2021. PMID: 33751579 Free PMC article. Review.
Gene therapy for inherited metabolic diseases.
Yilmaz BS, Gurung S, Perocheau D, Counsell J, Baruteau J. Yilmaz BS, et al. J Mother Child. 2020 Nov 10;24(2):53-64. doi: 10.34763/jmotherandchild.20202402si.2004.000009. J Mother Child. 2020. PMID: 33554501 Free PMC article. Review.
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. 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.
20 results