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Gene therapy for inherited metabolic diseases.
Yilmaz BS, Gurung S, Perocheau D, Counsell J, Baruteau J. Yilmaz BS, et al. Among authors: baruteau j. 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.
Clinical applications for exosomes: Are we there yet?
Perocheau D, Touramanidou L, Gurung S, Gissen P, Baruteau J. Perocheau D, et al. Among authors: baruteau j. 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.
Modelling urea cycle disorders using iPSCs.
Duff C, Baruteau J. Duff C, et al. Among authors: baruteau j. NPJ Regen Med. 2022 Sep 26;7(1):56. doi: 10.1038/s41536-022-00252-5. NPJ Regen Med. 2022. PMID: 36163209 Free PMC article. Review.
Safety and efficacy of an engineered hepatotropic AAV gene therapy for ornithine transcarbamylase deficiency in cynomolgus monkeys.
Baruteau J, Cunningham SC, Yilmaz BS, Perocheau DP, Eaglestone S, Burke D, Thrasher AJ, Waddington SN, Lisowski L, Alexander IE, Gissen P. Baruteau J, et al. Mol Ther Methods Clin Dev. 2021 Sep 14;23:135-146. doi: 10.1016/j.omtm.2021.09.005. eCollection 2021 Dec 10. Mol Ther Methods Clin Dev. 2021. PMID: 34703837 Free PMC article.
The incidence of movement disorder increases with age and contrasts with subtle and limited neuroimaging abnormalities in argininosuccinic aciduria.
Gurung S, Karamched S, Perocheau D, Seunarine KK, Baldwin T, Alrashidi H, Touramanidou L, Duff C, Elkhateeb N, Stepien KM, Sharma R, Morris A, Hartley T, Crowther L, Grunewald S, Cleary M, Mundy H, Chakrapani A, Batzios S, Davison J, Footitt E, Tuschl K, Lachmann R, Murphy E, Santra S, Uudelepp ML, Yeo M, Finn PF, Cavedon A, Siddiqui S, Rice L, Martini PGV, Frassetto A, Heales S, Mills PB, Gissen P, Clayden JD, Clark CA, Eaton S, Kalber TL, Baruteau J. Gurung S, et al. Among authors: baruteau j. J Inherit Metab Dis. 2023 Dec 4. doi: 10.1002/jimd.12691. Online ahead of print. J Inherit Metab Dis. 2023. PMID: 38044746
76 results