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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.
Worsening of cardiomyopathy using deflazacort in an animal model rescued by gene therapy.
Rotundo IL, Faraso S, De Leonibus E, Nigro G, Vitiello C, Lancioni A, Di Napoli D, Castaldo S, Russo V, Russo F, Piluso G, Auricchio A, Nigro V. Rotundo IL, et al. Among authors: de leonibus e. PLoS One. 2011;6(9):e24729. doi: 10.1371/journal.pone.0024729. Epub 2011 Sep 9. PLoS One. 2011. PMID: 21931833 Free PMC article.
Role of the dorsal hippocampus in object memory load.
Sannino S, Russo F, Torromino G, Pendolino V, Calabresi P, De Leonibus E. Sannino S, et al. Among authors: de leonibus e. Learn Mem. 2012 Apr 20;19(5):211-8. doi: 10.1101/lm.025213.111. Learn Mem. 2012. PMID: 22523415
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.
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.
68 results