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Fluoxetine ameliorates mucopolysaccharidosis type IIIA.
Capuozzo A, Montefusco S, Cacace V, Sofia M, Esposito A, Napolitano G, Nusco E, Polishchuk E, Pizzo MT, De Risi M, De Leonibus E, Sorrentino NC, Medina DL. Capuozzo A, et al. Among authors: de risi m, de leonibus e. Mol Ther. 2022 Apr 6;30(4):1432-1450. doi: 10.1016/j.ymthe.2022.01.037. Epub 2022 Feb 2. Mol Ther. 2022. PMID: 35121108 Free PMC article.
Long-term amelioration of feline Mucopolysaccharidosis VI after AAV-mediated liver gene transfer.
Cotugno G, Annunziata P, Tessitore A, O'Malley T, Capalbo A, Faella A, Bartolomeo R, O'Donnell P, Wang P, Russo F, Sleeper MM, Knox VW, Fernandez S, Levanduski L, Hopwood J, De Leonibus E, Haskins M, Auricchio A. Cotugno G, et al. Among authors: de leonibus e. Mol Ther. 2011 Mar;19(3):461-9. doi: 10.1038/mt.2010.257. Epub 2010 Nov 30. Mol Ther. 2011. PMID: 21119624 Free PMC article.
A highly secreted sulphamidase engineered to cross the blood-brain barrier corrects brain lesions of mice with mucopolysaccharidoses type IIIA.
Sorrentino NC, D'Orsi L, Sambri I, Nusco E, Monaco C, Spampanato C, Polishchuk E, Saccone P, De Leonibus E, Ballabio A, Fraldi A. Sorrentino NC, et al. Among authors: de leonibus e. EMBO Mol Med. 2013 May;5(5):675-90. doi: 10.1002/emmm.201202083. Epub 2013 Apr 9. EMBO Mol Med. 2013. PMID: 23568409 Free PMC article.
Lysosomal dysfunction disrupts presynaptic maintenance and restoration of presynaptic function prevents neurodegeneration in lysosomal storage diseases.
Sambri I, D'Alessio R, Ezhova Y, Giuliano T, Sorrentino NC, Cacace V, De Risi M, Cataldi M, Annunziato L, De Leonibus E, Fraldi A. Sambri I, et al. Among authors: de risi m, de leonibus e. EMBO Mol Med. 2017 Jan;9(1):112-132. doi: 10.15252/emmm.201606965. EMBO Mol Med. 2017. PMID: 27881461 Free PMC article.
AAV-mediated transcription factor EB (TFEB) gene delivery ameliorates muscle pathology and function in the murine model of Pompe Disease.
Gatto F, Rossi B, Tarallo A, Polishchuk E, Polishchuk R, Carrella A, Nusco E, Alvino FG, Iacobellis F, De Leonibus E, Auricchio A, Diez-Roux G, Ballabio A, Parenti G. Gatto F, et al. Among authors: de leonibus e. Sci Rep. 2017 Nov 8;7(1):15089. doi: 10.1038/s41598-017-15352-2. Sci Rep. 2017. PMID: 29118420 Free PMC article.
65 results