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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: Dawson, Glyn, Schroeder, Christina, Dawson, Philip E.
Formato: Artigo
Idioma:Inglês
Publicado: 2010
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Acceso en liña: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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