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-...
Main Authors: | , , , , , , , , , , , , |
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Format: | Journal article |
Language: | English |
Published: |
American Society for Clinical Investigation
2018
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