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Oxidative damage and cytotoxic cell involvement in the neuronal pathogenesis of mucopolysaccharidosis IIIB

Autor: VILLANI, GUGLIELMO ROSARIO DOMENI, DI NATALE, PAOLA, Di Domenico C., Musella A., Cecere F., Di Napoli D.

Mucopolysaccharidosis IIIB (MPS IIIB) is a lysosomal storage disease due to mutations in the gene encoding alpha-N-acetylglucosaminidase and is characterized by a severe neurological disorder. Although several studies have been reported for the murin

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=od______3730::ce7e8d3cc77531e7edba10321e794a72
http://hdl.handle.net/11588/353746

Lentiviral vector-mediated gene therapy for Sanfilippo B syndrome (MPS IIIB)

Autor: VILLANI, GUGLIELMO ROSARIO DOMENI, DI NATALE, PAOLA, DI DOMENICO C., GARGIULO N., FOLLENZI A., NALDINI L., DI NAPOLI D., CASTALDO S., GONZALEZ E., DE FELICE M., MITHBAOKAR P.

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=od______3730::10862d65267f498b13919ac709f69150
http://hdl.handle.net/11588/121975

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

Autor: Di Domenico C, Villani GR, Di Napoli D, Nusco E, Calì G, Nitsch L, Di Natale P.

Publikováno v: American journal of medical genetics. Part A 149A (2009): 1209–1218.
info:cnr-pdr/source/autori:Di Domenico C; Villani GR, Di Napoli D; Nusco E; Calì G; Nitsch L; Di Natale P./titolo:Intracranial gene delivery of LV-NAGLU vector corrects neuropathology in murine MPS IIIB./doi:/rivista:American journal of medical genetics. Part A/anno:2009/pagina_da:1209/pagina_a:1218/intervallo_pagine:1209–1218/volume:149A

Mucopolysacccharidosis (MPS) IIIB is an inherited lysosomal storage disorder caused by the deficiency of alpha-N-acetylglucosaminidase (NAGLU). The disease is characterized by mild somatic features and severe neurological involvement with high mortal

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=cnr_________::934824d882fd9ffdb8d1d980f7281b3a
http://www.cnr.it/prodotto/i/21191