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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: de leonibus e. 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.
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.
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.
miR-181a/b downregulation exerts a protective action on mitochondrial disease models.
Indrieri A, Carrella S, Romano A, Spaziano A, Marrocco E, Fernandez-Vizarra E, Barbato S, Pizzo M, Ezhova Y, Golia FM, Ciampi L, Tammaro R, Henao-Mejia J, Williams A, Flavell RA, De Leonibus E, Zeviani M, Surace EM, Banfi S, Franco B. Indrieri A, et al. Among authors: de leonibus e. EMBO Mol Med. 2019 May;11(5):e8734. doi: 10.15252/emmm.201708734. EMBO Mol Med. 2019. PMID: 30979712 Free PMC article.
65 results