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Three-Country Snapshot of Ornithine Transcarbamylase Deficiency.
Seker Yilmaz B, Baruteau J, Arslan N, Aydin HI, Barth M, Bozaci AE, Brassier A, Canda E, Cano A, Chronopoulou E, Connolly GM, Damaj L, Dawson C, Dobbelaere D, Douillard C, Eminoglu FT, Erdol S, Ersoy M, Fang S, Feillet F, Gokcay G, Goksoy E, Gorce M, Inci A, Kadioglu B, Kardas F, Kasapkara CS, Kilic Yildirim G, Kor D, Kose M, Marelli C, Mundy H, O'Sullivan S, Ozturk Hismi B, Ramachandran R, Roubertie A, Sanlilar M, Schiff M, Sreekantam S, Stepien KM, Uzun Unal O, Yildiz Y, Zubarioglu T, Gissen P. Seker Yilmaz B, et al. Among authors: gissen p. Life (Basel). 2022 Oct 27;12(11):1721. doi: 10.3390/life12111721. Life (Basel). 2022. PMID: 36362876 Free PMC article.
Delivering efficient liver-directed AAV-mediated gene therapy.
Baruteau J, Waddington SN, Alexander IE, Gissen P. Baruteau J, et al. Among authors: gissen p. Gene Ther. 2017 May;24(5):263-264. doi: 10.1038/gt.2016.90. Epub 2017 Jan 12. Gene Ther. 2017. PMID: 28079048 No abstract available.
Expanding the phenotype in argininosuccinic aciduria: need for new therapies.
Baruteau J, Jameson E, Morris AA, Chakrapani A, Santra S, Vijay S, Kocadag H, Beesley CE, Grunewald S, Murphy E, Cleary M, Mundy H, Abulhoul L, Broomfield A, Lachmann R, Rahman Y, Robinson PH, MacPherson L, Foster K, Chong WK, Ridout DA, Bounford KM, Waddington SN, Mills PB, Gissen P, Davison JE. Baruteau J, et al. Among authors: gissen p. J Inherit Metab Dis. 2017 May;40(3):357-368. doi: 10.1007/s10545-017-0022-x. Epub 2017 Mar 1. J Inherit Metab Dis. 2017. PMID: 28251416 Free PMC article.
Clinical applications for exosomes: Are we there yet?
Perocheau D, Touramanidou L, Gurung S, Gissen P, Baruteau J. Perocheau D, et al. Among authors: gissen p. 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.
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. Among authors: gissen p. 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.
192 results