Article
Fibroblast screening for chaperone therapy in beta-galactosidosis.
Brain & development - 1 Sept 2006
Iwasaki Hiroyuki, Watanabe Hiroshi, Iida Masami, Ogawa Seiichiro, Tabe Miho, Higaki Katsumi, Nanba Eiji, Suzuki Yoshiyuki
Abstract excerpt
We performed screening of beta-galactosidase-deficient fibroblasts for possible chemical chaperone therapy using N-octyl-4-epi-beta-valienamine (NOEV) in patients with GM1-gangliosidosis and Morquio B disease (beta-galactosidosis). Fibroblasts were cultured with NOEV for 4 days and beta-galactosidase activity was measured. Mutation analysis was performed simultaneously. Two separate criteria were set for...
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