Nusco E - Search Results

1

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: nusco e. Sci Rep. 2017 Nov 8;7(1):15089. doi: 10.1038/s41598-017-15352-2. Sci Rep. 2017. PMID: 29118420 Free PMC article.

2

SUMF1 enhances sulfatase activities in vivo in five sulfatase deficiencies.

Fraldi A, Biffi A, Lombardi A, Visigalli I, Pepe S, Settembre C, Nusco E, Auricchio A, Naldini L, Ballabio A, Cosma MP. Fraldi A, et al. Among authors: nusco e. Biochem J. 2007 Apr 15;403(2):305-12. doi: 10.1042/BJ20061783. Biochem J. 2007. PMID: 17206939 Free PMC article.

3

Systemic inflammation and neurodegeneration in a mouse model of multiple sulfatase deficiency.

Settembre C, Annunziata I, Spampanato C, Zarcone D, Cobellis G, Nusco E, Zito E, Tacchetti C, Cosma MP, Ballabio A. Settembre C, et al. Among authors: nusco e. Proc Natl Acad Sci U S A. 2007 Mar 13;104(11):4506-11. doi: 10.1073/pnas.0700382104. Epub 2007 Mar 6. Proc Natl Acad Sci U S A. 2007. PMID: 17360554 Free PMC article.

4

Disease rescue and increased lifespan in a model of cardiomyopathy and muscular dystrophy by combined AAV treatments.

Vitiello C, Faraso S, Sorrentino NC, Di Salvo G, Nusco E, Nigro G, Cutillo L, Calabrò R, Auricchio A, Nigro V. Vitiello C, et al. Among authors: nusco e. PLoS One. 2009;4(3):e5051. doi: 10.1371/journal.pone.0005051. Epub 2009 Mar 31. PLoS One. 2009. PMID: 19333401 Free PMC article.

5

Intracranial gene delivery of LV-NAGLU vector corrects neuropathology in murine MPS IIIB.

Di Domenico C, Villani GR, Di Napoli D, Nusco E, Calì G, Nitsch L, Di Natale P. Di Domenico C, et al. Among authors: nusco e. Am J Med Genet A. 2009 Jun;149A(6):1209-18. doi: 10.1002/ajmg.a.32861. Am J Med Genet A. 2009. PMID: 19449420

6

Correction of CNS defects in the MPSII mouse model via systemic enzyme replacement therapy.

Polito VA, Abbondante S, Polishchuk RS, Nusco E, Salvia R, Cosma MP. Polito VA, et al. Among authors: nusco e. Hum Mol Genet. 2010 Dec 15;19(24):4871-85. doi: 10.1093/hmg/ddq420. Epub 2010 Sep 27. Hum Mol Genet. 2010. PMID: 20876612 Free PMC article.

7

Efficacy of a combined intracerebral and systemic gene delivery approach for the treatment of a severe lysosomal storage disorder.

Spampanato C, De Leonibus E, Dama P, Gargiulo A, Fraldi A, Sorrentino NC, Russo F, Nusco E, Auricchio A, Surace EM, Ballabio A. Spampanato C, et al. Among authors: nusco e. Mol Ther. 2011 May;19(5):860-9. doi: 10.1038/mt.2010.299. Epub 2011 Feb 15. Mol Ther. 2011. PMID: 21326216 Free PMC article.

8

Impaired parkin-mediated mitochondrial targeting to autophagosomes differentially contributes to tissue pathology in lysosomal storage diseases.

de Pablo-Latorre R, Saide A, Polishhuck EV, Nusco E, Fraldi A, Ballabio A. de Pablo-Latorre R, et al. Among authors: nusco e. Hum Mol Genet. 2012 Apr 15;21(8):1770-81. doi: 10.1093/hmg/ddr610. Epub 2012 Jan 3. Hum Mol Genet. 2012. PMID: 22215441 Free PMC article.

9

Sustained reduction of hyperbilirubinemia in Gunn rats after adeno-associated virus-mediated gene transfer of bilirubin UDP-glucuronosyltransferase isozyme 1A1 to skeletal muscle.

Pastore N, Nusco E, Vaníkova J, Sepe RM, Vetrini F, McDonagh A, Auricchio A, Vitek L, Brunetti-Pierri N. Pastore N, et al. Among authors: nusco e. Hum Gene Ther. 2012 Oct;23(10):1082-9. doi: 10.1089/hum.2012.018. Epub 2012 Aug 27. Hum Gene Ther. 2012. PMID: 22765254

10

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: nusco 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.

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