Guaiacol as a drug candidate for treating adult polyglucosan body disease

Adult polyglucosan body disease (APBD) is a late-onset disease caused by intracellular accumulation of polyglucosan bodies, formed due to glycogen-branching enzyme (GBE) deficiency. To find a treatment for APBD, we screened 1,700 FDA-approved compounds in fibroblasts derived from APBD-modeling GBE1-...

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Опубликовано в: :JCI Insight
Главные авторы: Kakhlon, Or, Ferreira, Igor, Solmesky, Leonardo J., Khazanov, Netaly, Lossos, Alexander, Alvarez, Rafael, Yetil, Deniz, Pampou, Sergey, Weil, Miguel, Senderowitz, Hanoch, Escriba, Pablo, Yue, Wyatt W., Akman, H. Orhan
Формат: Artigo
Язык:Inglês
Опубликовано: American Society for Clinical Investigation 2018
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Research Article
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC6171812/
https://ncbi.nlm.nih.gov/pubmed/30185673
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/jci.insight.99694
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