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Full-length ATP7B reconstituted through protein trans-splicing corrects Wilson disease in mice.
Padula A, Petruzzelli R, Philbert SA, Church SJ, Esposito F, Campione S, Monti M, Capolongo F, Perna C, Nusco E, Schmidt HH, Auricchio A, Cooper GJS, Polishchuk R, Piccolo P. Padula A, et al. Among authors: nusco e. Mol Ther Methods Clin Dev. 2022 Aug 13;26:495-504. doi: 10.1016/j.omtm.2022.08.004. eCollection 2022 Sep 8. Mol Ther Methods Clin Dev. 2022. PMID: 36092366 Free PMC article.
Safe and effective liver-directed AAV-mediated homology-independent targeted integration in mouse models of inherited diseases.
Esposito F, Dell'Aquila F, Rhiel M, Auricchio S, Chmielewski KO, Andrieux G, Ferla R, Horrach PS, Padmanabhan A, Di Cunto R, Notaro S, Santeularia ML, Boerries M, Dell'Anno M, Nusco E, Padula A, Nutarelli S, Cornu TI, Sorrentino NC, Piccolo P, Trapani I, Cathomen T, Auricchio A. Esposito F, et al. Among authors: nusco e. Cell Rep Med. 2024 Jul 16;5(7):101619. doi: 10.1016/j.xcrm.2024.101619. Epub 2024 Jun 18. Cell Rep Med. 2024. PMID: 38897206 Free PMC article.
Liver gene therapy with intein-mediated F8 trans-splicing corrects mouse haemophilia A.
Esposito F, Lyubenova H, Tornabene P, Auricchio S, Iuliano A, Nusco E, Merlin S, Olgasi C, Manni G, Gargaro M, Fallarino F, Follenzi A, Auricchio A. Esposito F, et al. Among authors: nusco e. EMBO Mol Med. 2022 Jun 8;14(6):e15199. doi: 10.15252/emmm.202115199. Epub 2022 May 2. EMBO Mol Med. 2022. PMID: 35491676 Free PMC article.
Low incidence of hepatocellular carcinoma in mice and cats treated with systemic adeno-associated viral vectors.
Ferla R, Alliegro M, Dell'Anno M, Nusco E, Cullen JM, Smith SN, Wolfsberg TG, O'Donnell P, Wang P, Nguyen AD, Chandler RJ, Chen Z, Burgess SM, Vite CH, Haskins ME, Venditti CP, Auricchio A. Ferla R, et al. Among authors: nusco e. Mol Ther Methods Clin Dev. 2020 Nov 26;20:247-257. doi: 10.1016/j.omtm.2020.11.015. eCollection 2021 Mar 12. Mol Ther Methods Clin Dev. 2020. PMID: 33473358 Free PMC article.
46 results