Safety of arylsulfatase A overexpression for gene therapy of metachromatic leukodystrophy.

Capotondo, A; Cesani, M; Pepe, S; Fasano, S; Gregori, S; Tononi, L; Venneri, M A; Brambilla, R; Quattrini, A; Ballabio, A; Cosma, M P; Naldini, L; Biffi, A

Capotondo, A; Cesani, M; Pepe, S; Fasano, S; Gregori, S; Tononi, L; Venneri, M A; Brambilla, R; Quattrini, A; Ballabio, A; Cosma, M P; Naldini, L; Biffi, A.

Affiliation

Capotondo A; San Raffaele Telethon Institute for Gene Therapy, 20132 Milan, Italy.

Hum Gene Ther ; 18(9): 821-36, 2007 Sep.

Article in En | MEDLINE | ID: mdl-17845130

Subject(s)

Cerebroside-Sulfatase/metabolism; Genetic Therapy; Leukodystrophy, Metachromatic/genetics; Leukodystrophy, Metachromatic/therapy; Animals; Animals, Newborn; Antigens, CD34/immunology; Antigens, CD34/metabolism; Blotting, Southern; Cell Differentiation; Cell Lineage; Cell Proliferation; Cerebroside-Sulfatase/adverse effects; Cerebroside-Sulfatase/analysis; Colony-Forming Units Assay; Feasibility Studies; Genetic Vectors; Hematopoietic Stem Cells/cytology; Hematopoietic Stem Cells/metabolism; Humans; Lentivirus/genetics; Leukodystrophy, Metachromatic/metabolism; Leukodystrophy, Metachromatic/pathology; Mice; Mice, Transgenic; Models, Animal; Neurons/cytology; Neurons/metabolism; Polymerase Chain Reaction; Spleen/cytology; Transduction, Genetic

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Collection: 01-internacional Database: MEDLINE Main subject: Genetic Therapy / Cerebroside-Sulfatase / Leukodystrophy, Metachromatic Type of study: Prognostic_studies Limits: Animals / Humans Language: En Journal: Hum Gene Ther Journal subject: GENETICA MEDICA / TERAPEUTICA Year: 2007 Type: Article Affiliation country: Italy

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