Prediction of the responsiveness to pharmacological chaperones: lysosomal human alpha-galactosidase, a case of study

BACKGROUND: The pharmacological chaperones therapy is a promising approach to cure genetic diseases. It relies on substrate competitors used at sub-inhibitory concentration which can be administered orally, reach difficult tissues and have low cost. Clinical trials are currently carried out for Fabr...

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Hlavní autoři: Andreotti, Giuseppina, Guarracino, Mario R, Cammisa, Marco, Correra, Antonella, Cubellis, Maria Vittoria
Médium: Artigo
Jazyk:Inglês
Vydáno: BioMed Central 2010
Témata:
Methodology
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC3016270/
https://ncbi.nlm.nih.gov/pubmed/21138548
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/1750-1172-5-36
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