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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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| Main Authors: | , , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado: |
BioMed Central
2010
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| Assuntos: | |
| Acceso en liña: | 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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