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Identification and Characterization of Ambroxol as an Enzyme Enhancement Agent for Gaucher Disease

Gaucher disease (GD), the most prevalent lysosomal storage disease, is caused by a deficiency of glucocerebrosidase (GCase). The identification of small molecules acting as agents for enzyme enhancement therapy is an attractive approach for treating different forms of GD. A thermal denaturation assa...

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Detalhes bibliográficos
Main Authors: Maegawa, Gustavo H. B., Tropak, Michael B., Buttner, Justin D., Rigat, Brigitte A., Fuller, Maria, Pandit, Deepangi, Tang, Liangiie, Kornhaber, Gregory J., Hamuro, Yoshitomo, Clarke, Joe T. R., Mahuran, Don J.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society for Biochemistry and Molecular Biology 2009
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC2749124/
https://ncbi.nlm.nih.gov/pubmed/19578116
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1074/jbc.M109.012393
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