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139 results

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Page 1
Retinal gene therapy for Stargardt disease with dual AAV intein vectors is both safe and effective in large animal models.
Ferla R, Pugni E, Lupo M, Tiberi P, Fioretto F, Perota A, Duchi R, Lagutina I, Gesualdo C, Rossi S, Ventrella D, Elmi A, McClinton B, Toomes C, Xu T, Molday RS, Surace EM, Simonelli F, Bacci ML, Galli C, Memon MA, Shams N, Auricchio A, Trapani I. Ferla R, et al. Among authors: auricchio a. Sci Adv. 2025 Mar 28;11(13):eadt9354. doi: 10.1126/sciadv.adt9354. Epub 2025 Mar 26. Sci Adv. 2025. PMID: 40138422 Free PMC article.
Multi-year enzyme expression in patients with mucopolysaccharidosis type VI after liver-directed gene therapy.
Rossi A, Romano R, Fecarotta S, Dell'Anno M, Pecorella V, Passeggio R, Zancan S, Parenti G, Santamaria F, Borgia F, Deodato F, Funghini S, Rupar CA, Prasad C, O'Callaghan M, Mitchell JJ, Valsecchi MG, la Marca G, Galimberti S, Auricchio A, Brunetti-Pierri N. Rossi A, et al. Among authors: auricchio a. Med. 2025 Apr 11;6(4):100544. doi: 10.1016/j.medj.2024.10.021. Epub 2024 Nov 14. Med. 2025. PMID: 39547230 Clinical Trial.
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: auricchio a. 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.
Multicentric Longitudinal Prospective Study in a European Cohort of MYO7A Patients: Disease Course and Implications for Gene Therapy.
Testa F, Carreño E, van den Born LI, Melillo P, Perea-Romero I, Di Iorio V, Risca G, Iodice CM, Pennings RJE, Karali M, Banfi S, Auricchio A, Galimberti S, Ayuso C, Simonelli F. Testa F, et al. Among authors: auricchio a. Invest Ophthalmol Vis Sci. 2024 Jun 3;65(6):25. doi: 10.1167/iovs.65.6.25. Invest Ophthalmol Vis Sci. 2024. PMID: 38884554 Free PMC article.
Efficacy, pharmacokinetics, and safety in the mouse and primate retina of dual AAV vectors for Usher syndrome type 1B.
Ferla R, Dell'Aquila F, Doria M, Ferraiuolo M, Noto A, Grazioli F, Ammendola V, Testa F, Melillo P, Iodice C, Risca G, Tedesco N, le Brun PR, Surace EM, Simonelli F, Galimberti S, Valsecchi MG, Marteau JB, Veron P, Colloca S, Auricchio A. Ferla R, et al. Among authors: auricchio a. Mol Ther Methods Clin Dev. 2023 Feb 11;28:396-411. doi: 10.1016/j.omtm.2023.02.002. eCollection 2023 Mar 9. Mol Ther Methods Clin Dev. 2023. PMID: 36910588 Free PMC article.
Liver-directed gene therapy for ornithine aminotransferase deficiency.
Boffa I, Polishchuk E, De Stefano L, Dell'Aquila F, Nusco E, Marrocco E, Audano M, Pedretti S, Caterino M, Bellezza I, Ruoppolo M, Mitro N, Cellini B, Auricchio A, Brunetti-Pierri N. Boffa I, et al. Among authors: auricchio a. EMBO Mol Med. 2023 Apr 11;15(4):e17033. doi: 10.15252/emmm.202217033. Epub 2023 Jan 17. EMBO Mol Med. 2023. PMID: 36647689 Free PMC article.
miR-181a/b downregulation: a mutation-independent therapeutic approach for inherited retinal diseases.
Carrella S, Di Guida M, Brillante S, Piccolo D, Ciampi L, Guadagnino I, Garcia Piqueras J, Pizzo M, Marrocco E, Molinari M, Petrogiannakis G, Barbato S, Ezhova Y, Auricchio A, Franco B, De Leonibus E, Surace EM, Indrieri A, Banfi S. Carrella S, et al. Among authors: auricchio a. EMBO Mol Med. 2022 Nov 8;14(11):e15941. doi: 10.15252/emmm.202215941. Epub 2022 Oct 4. EMBO Mol Med. 2022. PMID: 36194668 Free PMC article.
139 results