Intracranial gene delivery of LV-NAGLU vector corrects neuropathology in murine MPS IIIB. (Articolo in rivista)

Type

• Prodotto della ricerca (Classe)
• Articolo in rivista (Classe)

Label

• Intracranial gene delivery of LV-NAGLU vector corrects neuropathology in murine MPS IIIB. (Articolo in rivista) (literal)

Anno

• 2009-01-01T00:00:00+01:00 (literal)

Alternative label

• Di Domenico C; Villani GR, Di Napoli D; Nusco E; Calì G; Nitsch L; Di Natale P. (2009)
  Intracranial gene delivery of LV-NAGLU vector corrects neuropathology in murine MPS IIIB.
  in American journal of medical genetics. Part A
  (literal)

Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#autori

• Di Domenico C; Villani GR, Di Napoli D; Nusco E; Calì G; Nitsch L; Di Natale P. (literal)

Pagina inizio

• 1209 (literal)

Pagina fine

• 1218 (literal)

Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#numeroVolume

• 149A (literal)

Rivista

• American journal of medical genetics. Part A (Rivista)

Note

• ISI Web of Science (WOS) (literal)

Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#affiliazioni

• Univ Naples Federico 2, Dept Biochem & Med Biotechnol, I-80131 Naples, Italy CEINGE Adv Biotechnol, Naples, Italy Cardarelli Hosp, Ctr Anim Experimentat, Naples, Italy TIGEM, Naples, Italy IEOS G Salvatore Natl Council Res, Naples, Italy Univ Naples Federico 2, Dept Mol & Cellular Biol & Pathol, I-80131 Naples, Italy (literal)

Titolo

• Intracranial gene delivery of LV-NAGLU vector corrects neuropathology in murine MPS IIIB. (literal)

Abstract

• 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 mortality. Although several therapeutic approaches have been applied to the murine model of the disease, no effective therapy is available for patients. In this study, we used the lentiviral-NAGLU vector to deliver the functional human NAGLU gene into the brain of young adult MPS IIIB mice. We report the restoration of active enzyme with a sustained expression throughout a large portion of the brain, and a significantly improved behavioral performance of treated animals. Moreover, we analyzed the effect of therapy on the expression profile of some genes related to neurotrophic signaling molecules and inflammatory cytokines previously found altered in MPS IIIB mice. At 1 month from treatment, the level of cerebellin 1 (Cbln1) was decreased while the brain-derived neurotrophic factor (Bdnf) expression was increased, both reaching normal values. At 6 months from treatment a significant reduction in the expression of all the inflammation- and oxidative stress-related genes was observed, as well as the maintenance of the correction of the Bdnf gene expression. These results indicate that NAGLU delivery from intracerebral sources has the capacity to alleviate most disease manifestations in MPS IIIB mice; furthermore, Bdnf might be a response-to-therapy biomarker for MPS IIIB. (literal)

Prodotto di

• Identificazione e caratterizzazione funzionale dei geni e delle proteine coinvolti nella tumorigenesi tiroidea (SV.P15.001.004) (Modulo)
• Institute Experimental Endocrinology and Oncology \"Gaetano Salvatore\" (IEOS) (Istituto)

Autore CNR

• GAETANO CALI' (Unità di personale interno)

Incoming links:

Autore CNR di

• GAETANO CALI' (Unità di personale interno)

Prodotto

• Institute Experimental Endocrinology and Oncology \"Gaetano Salvatore\" (IEOS) (Istituto)
• Identificazione e caratterizzazione funzionale dei geni e delle proteine coinvolti nella tumorigenesi tiroidea (SV.P15.001.004) (Modulo)

Http://www.cnr.it/ontology/cnr/pubblicazioni.owl#rivistaDi

• American journal of medical genetics. Part A (Rivista)