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Guaiacol as a drug candidate for treating adult polyglucosan body disease

Abstract:

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-knockin mice. Capitalizing on fluorescent periodic acid–Schiff reagent, which interacts with polyglucosans in the cell, this screen discovered that the flavoring agent guaiacol can lower polyglucos...

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Publication status:
Published
Peer review status:
Peer reviewed

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Publisher copy:
10.1172/jci.insight.99694

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Publisher:
American Society for Clinical Investigation Publisher's website
Journal:
JCI Insight Journal website
Volume:
3
Issue:
17
Article number:
e99694
Publication date:
2018-09-06
Acceptance date:
2018-07-31
DOI:
ISSN:
2379-3708
Pmid:
30185673
Source identifiers:
915399
Language:
English
Keywords:
Pubs id:
pubs:915399
UUID:
uuid:cf252429-af8f-4e78-8c32-b469f3069d0f
Local pid:
pubs:915399
Deposit date:
2018-10-10

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