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Getting into the fold

Pharmacological chaperones are small molecules that interact with a protein to enhance folding and stability. Understanding the chemistry underlying the interaction of pharmacological chaperones with their target proteins will facilitate the design of more efficient drugs with greater therapeutic potential.

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Figure 1: The action of pharmacological chaperones.

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I have nothing to declare in relation to Gaucher disease, but do receive funding from a company for research purposes, on chemical chaperones.

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Brooks, D. Getting into the fold. Nat Chem Biol 3, 84–85 (2007). https://doi.org/10.1038/nchembio0207-84

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