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Liver-directed gene therapy for inherited metabolic diseases.
Baruteau J, Brunetti-Pierri N, Gissen P. Baruteau J, et al. Among authors: brunetti pierri n. J Inherit Metab Dis. 2024 Jan;47(1):9-21. doi: 10.1002/jimd.12709. Epub 2024 Jan 3. J Inherit Metab Dis. 2024. PMID: 38171926 Review.
A retrograde approach for liver gene transfer.
Brunetti-Pierri N, Gissen P. Brunetti-Pierri N, et al. Mol Ther Methods Clin Dev. 2022 Nov 25;27:488-490. doi: 10.1016/j.omtm.2022.11.002. eCollection 2022 Dec 8. Mol Ther Methods Clin Dev. 2022. PMID: 36458113 Free PMC article. No abstract available.
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.
Gene therapies for mucopolysaccharidoses.
Rossi A, Brunetti-Pierri N. Rossi A, et al. J Inherit Metab Dis. 2024 Jan;47(1):135-144. doi: 10.1002/jimd.12626. Epub 2023 May 29. J Inherit Metab Dis. 2024. PMID: 37204267 Review.
Hepatic glutamine synthetase augmentation enhances ammonia detoxification.
Soria LR, Nitzahn M, De Angelis A, Khoja S, Attanasio S, Annunziata P, Palmer DJ, Ng P, Lipshutz GS, Brunetti-Pierri N. Soria LR, et al. J Inherit Metab Dis. 2019 Nov;42(6):1128-1135. doi: 10.1002/jimd.12070. Epub 2019 Mar 11. J Inherit Metab Dis. 2019. PMID: 30724386 Free PMC article.
259 results