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The Amyloid Inhibitor CLR01 Relieves Autophagy and Ameliorates Neuropathology in a Severe Lysosomal Storage Disease.
Monaco A, Maffia V, Sorrentino NC, Sambri I, Ezhova Y, Giuliano T, Cacace V, Nusco E, De Risi M, De Leonibus E, Schrader T, Klärner FG, Bitan G, Fraldi A. Monaco A, et al. Among authors: de leonibus e. Mol Ther. 2022 Nov 2;30(11):3499. doi: 10.1016/j.ymthe.2022.10.001. Epub 2022 Oct 18. Mol Ther. 2022. PMID: 36261045 Free PMC article. No abstract available.
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.
Enhancing the Therapeutic Potential of Sulfamidase for the Treatment of Mucopolysaccharidosis IIIA.
Sorrentino NC, Cacace V, De Risi M, Maffia V, Strollo S, Tedesco N, Nusco E, Romagnoli N, Ventrella D, Huang Y, Liu N, Kalled SL, Choi VW, De Leonibus E, Fraldi A. Sorrentino NC, et al. Among authors: de risi m, de leonibus e. Mol Ther Methods Clin Dev. 2019 Oct 28;15:333-342. doi: 10.1016/j.omtm.2019.10.009. eCollection 2019 Dec 13. Mol Ther Methods Clin Dev. 2019. PMID: 31788497 Free PMC article.
The Amyloid Inhibitor CLR01 Relieves Autophagy and Ameliorates Neuropathology in a Severe Lysosomal Storage Disease.
Monaco A, Maffia V, Sorrentino NC, Sambri I, Ezhova Y, Giuliano T, Cacace V, Nusco E, De Risi M, De Leonibus E, Schrader T, Klärner FG, Bitan G, Fraldi A. Monaco A, et al. Among authors: de risi m, de leonibus e. Mol Ther. 2020 Apr 8;28(4):1167-1176. doi: 10.1016/j.ymthe.2020.02.005. Epub 2020 Feb 12. Mol Ther. 2020. PMID: 32087148 Free PMC article.
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.
Altered heparan sulfate metabolism during development triggers dopamine-dependent autistic-behaviours in models of lysosomal storage disorders.
De Risi M, Tufano M, Alvino FG, Ferraro MG, Torromino G, Gigante Y, Monfregola J, Marrocco E, Pulcrano S, Tunisi L, Lubrano C, Papy-Garcia D, Tuchman Y, Salleo A, Santoro F, Bellenchi GC, Cristino L, Ballabio A, Fraldi A, De Leonibus E. De Risi M, et al. Among authors: de leonibus e. Nat Commun. 2021 Jun 9;12(1):3495. doi: 10.1038/s41467-021-23903-5. Nat Commun. 2021. PMID: 34108486 Free PMC article.
65 results