Biochem/physiol Actions
AN1 is a brain blood barrier penetrant agent that tether LC3 and mHTT and targets mHTT for autophagosome degradation. AN1 significantly lower mHTT in cortices of HD mice, in cells from patients with Huntington’;s disease, and neurons derived from induced pluripotent stem cells. AN1 does not lower wtHTT in fibroblasts from healthy human donors. It appears that AN1 interact with the polyQ stretch.
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