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Titolo:
Primary adult human astrocytes as an ex vivo vehicle for beta-glucuronidase delivery in the brain
Autore:
Serguera, C; Sarkis, C; Ridet, JL; Colin, P; Moullier, P; Mallet, J;
Indirizzi:
Hop La Pitie Salpetriere, Lab Genet Mol Neurotransmiss & Proc Neurodegenera, CNRS UMR 9923, F-75013 Paris, France Hop La Pitie Salpetriere Paris France F-75013 923, F-75013 Paris, France CHU Hotel Dieu, Lab Therapie Gen, F-44000 Nantes, France CHU Hotel Dieu Nantes France F-44000 herapie Gen, F-44000 Nantes, France
Titolo Testata:
MOLECULAR THERAPY
fascicolo: 6, volume: 3, anno: 2001,
pagine: 875 - 881
SICI:
1525-0016(200106)3:6<875:PAHAAA>2.0.ZU;2-G
Fonte:
ISI
Lingua:
ENG
Soggetto:
MUCOPOLYSACCHARIDOSIS TYPE-VII; LYSOSOMAL STORAGE DISEASE; RECOMBINANT ADENOASSOCIATED VIRUS; MEDIATED GENE-TRANSFER; CENTRAL-NERVOUS-SYSTEM; MURINE MODEL; MOUSE-BRAIN; IN-VITRO; MICE; PATHOLOGY;
Keywords:
beta-glucuronidase; human astrocytes; gene therapy; neurotransplantation;
Tipo documento:
Article
Natura:
Periodico
Settore Disciplinare:
Life Sciences
Citazioni:
37
Recensione:
Indirizzi per estratti:
Indirizzo: Mallet, J Hop La Pitie Salpetriere, Lab Genet Mol Neurotransmiss & Proc Neurodegenera, CNRS UMR 9923, Batiment CERVI,83 Blvd Hop, F-75013 Paris, France Hop La Pitie Salpetriere Batiment CERVI,83 Blvd Hop Paris France F-75013
Citazione:
C. Serguera et al., "Primary adult human astrocytes as an ex vivo vehicle for beta-glucuronidase delivery in the brain", MOL THER, 3(6), 2001, pp. 875-881

Abstract

Astrocytes are a good candidate cell type for brain transplantation: They are endogenous to the CNS, they have efficient secretory machinery, and they play a major role in neuronal support. We assessed the potential of genetically modified primary adult human astrocytes as vehicles for the deliveryof secreted molecules in the mammalian CNS. We report that such cells can be efficiently transduced by a recombinant adenoviral vector carrying the human beta -glucuronidase cDNA (Ad/ CMV*beta -glu) and that the transduced astrocytes produce large amounts of the enzyme. Released beta -glucuronidasecould be captured, in vitro, by primary neurons and astrocytes and by a neuroblastoma cell line and beta -glucuronidase-deficient fibroblasts. Following grafting into the mouse striatum, adult human astrocytes survived and expressed the transgene for at least 8 weeks. Moreover, the dosage of beta -glucuronidase activity within the grafted brains revealed high enzymatic levels at a long distance from the graft. These experiments document the grafting of engineered primary adult human astrocytes, allowing the release of a secreted therapeutic factor throughout the brain.

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Documento generato il 21/10/20 alle ore 11:14:31