Chemical chaperone therapy for brain pathology in G(M1)-gangliosidosis

We synthesized a galactose derivative, N-octyl-4-epi-β-valienamine (NOEV), for a molecular therapy (chemical chaperone therapy) of a human neurogenetic disease, β-galactosidosis (G(M1)-gangliosidosis and Morquio B disease). It is a potent inhibitor of lysosomal β-galactosidase in vitro. Addition of...

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Detalhes bibliográficos
Main Authors: Matsuda, Junichiro, Suzuki, Osamu, Oshima, Akihiro, Yamamoto, Yoshie, Noguchi, Akira, Takimoto, Kazuhiro, Itoh, Masayuki, Matsuzaki, Yuji, Yasuda, Yosuke, Ogawa, Seiichiro, Sakata, Yuko, Nanba, Eiji, Higaki, Katsumi, Ogawa, Yoshimi, Tominaga, Lika, Ohno, Kousaku, Iwasaki, Hiroyuki, Watanabe, Hiroshi, Brady, Roscoe O., Suzuki, Yoshiyuki
Formato: Artigo
Idioma:Inglês
Publicado em: National Academy of Sciences 2003
Assuntos:
Biological Sciences
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC307667/
https://ncbi.nlm.nih.gov/pubmed/14676316
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.2536657100
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