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HTS strategies for identifying compounds enhancing the activity of mutant lysosomal enzymes.
| Assay | Target | Readout | Compound classes |
|---|---|---|---|
| Inhibitors | Purified protein | Decreased enzyme activity | ASCC |
| Attenuators of denaturation | Purified protein | Increased residual enzyme activity following heat denaturation | ASCC, SCC |
| Residual enzyme activity enhancers | Live cells | Increased enzyme activity in cell lysates | ASCC, SCC, alternative pathway enhancers |
