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Liver-Directed Adeno-Associated Virus-Mediated Gene Therapy for Mucopolysaccharidosis Type VI.
Brunetti-Pierri N, Ferla R, Ginocchio VM, Rossi A, Fecarotta S, Romano R, Parenti G, Yildiz Y, Zancan S, Pecorella V, Dell'Anno M, Graziano M, Alliegro M, Andria G, Santamaria F, Brunetti-Pierri R, Simonelli F, Nigro V, Vargas M, Servillo G, Borgia F, Soscia E, Gargaro M, Funghini S, Tedesco N, Le Brun PR, Rupar CA, Prasad C, O'Callaghan M, Mitchell JJ, Danos O, Marteau JB, Galimberti S, Valsecchi MG, Veron P, Mingozzi F, Fallarino F, la Marca G, Sivri HS, Auricchio A. Brunetti-Pierri N, et al. Among authors: simonelli f. NEJM Evid. 2022 Jul;1(7):EVIDoa2200052. doi: 10.1056/EVIDoa2200052. Epub 2022 Jun 6. NEJM Evid. 2022. PMID: 38319253
Metabolomic fingerprinting of renal disease progression in Bardet-Biedl syndrome reveals mitochondrial dysfunction in kidney tubular cells.
Marchese E, Caterino M, Viggiano D, Cevenini A, Tolone S, Docimo L, Di Iorio V, Del Vecchio Blanco F, Fedele R, Simonelli F, Perna A, Nigro V, Capasso G, Ruoppolo M, Zacchia M. Marchese E, et al. Among authors: simonelli f. iScience. 2022 Sep 27;25(11):105230. doi: 10.1016/j.isci.2022.105230. eCollection 2022 Nov 18. iScience. 2022. PMID: 36281451 Free PMC article.
Ocular pharmacological and biochemical profiles of 6-thioguanine: a drug repurposing study.
Trotta MC, Gesualdo C, Lepre CC, Russo M, Ferraraccio F, Panarese I, Marano E, Grieco P, Petrillo F, Hermenean A, Simonelli F, D'Amico M, Bucolo C, Lazzara F, De Nigris F, Rossi S, Platania CBM. Trotta MC, et al. Among authors: simonelli f. Front Pharmacol. 2024 Mar 25;15:1375805. doi: 10.3389/fphar.2024.1375805. eCollection 2024. Front Pharmacol. 2024. PMID: 38590636 Free PMC article.
Inhibition of Galectins and the P2X7 Purinergic Receptor as a Therapeutic Approach in the Neurovascular Inflammation of Diabetic Retinopathy.
Lepre CC, Russo M, Trotta MC, Petrillo F, D'Agostino FA, Gaudino G, D'Amico G, Campitiello MR, Crisci E, Nicoletti M, Gesualdo C, Simonelli F, D'Amico M, Hermenean A, Rossi S. Lepre CC, et al. Among authors: simonelli f. Int J Mol Sci. 2023 Jun 3;24(11):9721. doi: 10.3390/ijms24119721. Int J Mol Sci. 2023. PMID: 37298672 Free PMC article. Review.
Voretigene neparvovec for inherited retinal dystrophy due to RPE65 mutations: a scoping review of eligibility and treatment challenges from clinical trials to real practice.
Testa F, Bacci G, Falsini B, Iarossi G, Melillo P, Mucciolo DP, Murro V, Salvetti AP, Sodi A, Staurenghi G, Simonelli F. Testa F, et al. Among authors: simonelli f. Eye (Lond). 2024 Apr 16. doi: 10.1038/s41433-024-03065-6. Online ahead of print. Eye (Lond). 2024. PMID: 38627549 Review.
252 results