AAV8-mediated expression of glucocerebrosidase ameliorates the storage pathology in the visceral organs of a mouse model of Gaucher disease.

McEachern, Kerry Anne; Nietupski, Jennifer B; Chuang, Wei-Lien; Armentano, Donna; Johnson, Jennifer; Hutto, Elizabeth; Grabowski, Gregory A; Cheng, Seng H; Marshall, John

McEachern, Kerry Anne; Nietupski, Jennifer B; Chuang, Wei-Lien; Armentano, Donna; Johnson, Jennifer; Hutto, Elizabeth; Grabowski, Gregory A; Cheng, Seng H; Marshall, John.

Affiliation

McEachern KA; Genzyme Corporation, 31 New York Avenue, Framingham, MA 01701-9322, USA.

J Gene Med ; 8(6): 719-29, 2006 Jun.

Article in En | MEDLINE | ID: mdl-16528760

Subject(s)

Dependovirus/genetics; Gaucher Disease/enzymology; Gaucher Disease/pathology; Genetic Therapy; Glucosylceramidase/genetics; Glucosylceramidase/metabolism; Viscera/pathology; Animals; Bronchoalveolar Lavage Fluid/cytology; Disease Models, Animal; Dose-Response Relationship, Drug; Gaucher Disease/genetics; Gaucher Disease/therapy; Gene Expression Regulation, Enzymologic; Humans; Immune Tolerance/immunology; Liver/cytology; Liver/pathology; Lung/cytology; Lung/pathology; Macrophages/cytology; Macrophages/pathology; Mice; Mice, Inbred C57BL; Spleen/cytology; Spleen/pathology; Treatment Outcome

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Collection: 01-internacional Database: MEDLINE Main subject: Viscera / Genetic Therapy / Dependovirus / Gaucher Disease / Glucosylceramidase Limits: Animals / Humans Language: En Journal: J Gene Med Journal subject: BIOLOGIA MOLECULAR / GENETICA MEDICA Year: 2006 Document type: Article Affiliation country: United States Country of publication: United kingdom

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