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Anti-amyloid treatment is broadly effective in neuronopathic mucopolysaccharidoses and synergizes with gene therapy in MPS-IIIA.
Giaccio M, Monaco A, Galiano L, Parente A, Borzacchiello L, Rubino R, Klärner FG, Killa D, Perna C, Piccolo P, Marotta M, Pan X, Khijniak M, Siddique I, Schrader T, Pshezhetsky AV, Sorrentino NC, Bitan G, Fraldi A. Giaccio M, et al. Among authors: piccolo p. Mol Ther. 2024 Nov 6;32(11):4108-4121. doi: 10.1016/j.ymthe.2024.09.030. Epub 2024 Sep 28. Mol Ther. 2024. PMID: 39342429 Free PMC article.
Sestrin2 drives ER-phagy in response to protein misfolding.
De Leonibus C, Maddaluno M, Ferriero R, Besio R, Cinque L, Lim PJ, Palma A, De Cegli R, Gagliotta S, Montefusco S, Iavazzo M, Rohrbach M, Giunta C, Polishchuk E, Medina DL, Di Bernardo D, Forlino A, Piccolo P, Settembre C. De Leonibus C, et al. Among authors: piccolo p. Dev Cell. 2024 Aug 19;59(16):2035-2052.e10. doi: 10.1016/j.devcel.2024.07.004. Epub 2024 Aug 1. Dev Cell. 2024. PMID: 39094564 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: piccolo p. 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.
Identification of hemodynamically stable patients with acute pulmonary embolism at high risk for death: external validation of different models.
Becattini C, Vedovati MC, Colombo S, Vanni S, Abrignani MG, Scardovi AB, Marrazzo A, Borselli M, Barchetti M, Fabbri A, Dentali F, Maggioni AP, Agnelli G, Gulizia MM; COntemporary management of Pulmonary Embolism (COPE) Investigators. Becattini C, et al. J Thromb Haemost. 2024 Sep;22(9):2502-2513. doi: 10.1016/j.jtha.2024.04.025. Epub 2024 May 27. J Thromb Haemost. 2024. PMID: 38810699 Free article.
104 results