Gene therapy of Hunter syndrome: evaluation of the efficiency of muscle electro gene transfer for the production and release of recombinant iduronate-2-sulfatase (IDS).

Friso, A; Tomanin, R; Zanetti, A; Mennuni, C; Calvaruso, F; La Monica, N; Marin, O; Zacchello, F; Scarpa, M

Friso, A; Tomanin, R; Zanetti, A; Mennuni, C; Calvaruso, F; La Monica, N; Marin, O; Zacchello, F; Scarpa, M.

Affiliation

Friso A; Gene Therapy Laboratory, Centre for Rare Diseases and Dept of Pediatrics, University of Padova, Via Giustiniani 3, 35128 Padova, Italy.

Biochim Biophys Acta ; 1782(10): 574-80, 2008 Oct.

Article in En | MEDLINE | ID: mdl-18675343

Subject(s)

Genetic Therapy/methods; Glycoproteins/metabolism; Mucopolysaccharidosis II/therapy; Quadriceps Muscle/metabolism; Animals; Antibody Formation/immunology; Cattle; Electric Stimulation/methods; Feasibility Studies; Glycoproteins/genetics; Glycoproteins/immunology; Glycosaminoglycans/metabolism; Glycosaminoglycans/pharmacology; Humans; Hyaluronoglucosaminidase/metabolism; Mice; Mice, Inbred C57BL; Mice, Knockout; Mucopolysaccharidosis II/genetics; Quadriceps Muscle/drug effects; Recombinant Proteins/genetics; Recombinant Proteins/immunology; Recombinant Proteins/metabolism

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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Glycoproteins / Genetic Therapy / Mucopolysaccharidosis II / Quadriceps Muscle Type of study: Guideline / Prognostic_studies Limits: Animals / Humans Language: En Journal: Biochim Biophys Acta Year: 2008 Document type: Article Affiliation country: Italy Country of publication: Netherlands

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