Transient high-level expression of ß-galactosidase after transfection of fibroblasts from GM1 gangliosidosis patients with plasmid DNA

Transient high-level expression of ß-galactosidase after transfection of fibroblasts from GM1 gangliosidosis patients with plasmid DNA

GM1 gangliosidosis is an autosomal recessive disorder caused by the deficiency of lysosomal acid hydrolase ß-galactosidase (ß-Gal). It is one of the most frequent lysosomal storage disorders in Brazil, with an estimated frequency of 1:17,000. The enzyme is secreted and can be captured by deficient c...

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Bibliographic Details
Main Authors: R.C. Balestrin, G. Baldo, M.B. Vieira, R. Sano, J.C. Coelho, R. Giugliani, U. Matte
Format: Article
Language:English
Published: Associação Brasileira de Divulgação Científica 2008-04-01
Series:Brazilian Journal of Medical and Biological Research
Subjects:
GM1 gangliosidosis
ß-galactosidase deficiency
Gene therapy
Lysosomal storage disorder
Lipofectamine
Online Access:http://www.scielo.br/scielo.php?script=sci_arttext&pid=S0100-879X2008000400005

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http://www.scielo.br/scielo.php?script=sci_arttext&pid=S0100-879X2008000400005

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