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...

Cijeli opis

Bibliografski detalji

Glavni autori:	R.C. Balestrin, G. Baldo, M.B. Vieira, R. Sano, J.C. Coelho, R. Giugliani, U. Matte
Format:	Artigo
Jezik:	Inglês
Izdano:	Associação Brasileira de Divulgação Científica 2008-04-01
Serija:	Brazilian Journal of Medical and Biological Research
Teme:	GM1 gangliosidosis ß-galactosidase deficiency Gene therapy Lysosomal storage disorder Lipofectamine
Online pristup:	http://www.scielo.br/scielo.php?script=sci_arttext&pid=S0100-879X2008000400005
Oznake:	Dodaj oznaku Bez oznaka, Budi prvi tko označuje ovaj zapis!