Figure 8. Probe compound ML247 restores GAA translocation to the lysosome (blue arrows).

Figure 8Probe compound ML247 restores GAA translocation to the lysosome (blue arrows)

False-colored images of fibroblast cells show DAPI nuclear stain (blue), the lysosomal marker Cathepsin D (red) and GAA (green) in cells from (A) an unaffected donor with wildtype GAA, and (B-E) Pompe patient-derived cells with p.Y455C/p.G638W mutations. Cells were cultured for five days with compound that was dispensed using a DMSO vehicle. Yellow color (overlapping green and red) indicates co-localized lysosomal GAA protein, typically present in (A) DMSO vehicle treated wildtype cells, but absent in (B) DMSO vehicle treated patient-derived cells, and partially restored with (C) 20 μM duvoglustat, (D) 15μM compound 1, and more strongly with (E) 15 μM compound 16.

From: Discovery, SAR, and Biological Evaluation of a Non-Inhibitory Chaperone for Acid Alpha Glucosidase

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Probe Reports from the NIH Molecular Libraries Program [Internet].
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