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Palmitoyl:protein thioesterase (PPT1) inhibitors can act as pharmacological chaperones in infantile Batten Disease
Competitive inhibitors of lysosomal hydrolases (pharmacological chaperones) have been used to treat some lysosomal storage diseases which result from mis-sense mutations and misfolded protein but have not been tried in Batten disease, for which there is no current therapy. We synthesized a large num...
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| Main Authors: | , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2010
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC2881699/ https://ncbi.nlm.nih.gov/pubmed/20346914 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.bbrc.2010.03.137 |
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