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

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http://www.cnr.it/prodotto/i/21191