A new study fulfills a central goal of post-genomic medicine, the treatment of inherited loss-of-function disorders not by correcting a genetic mutation but by augmenting the efficiency with which the nascent mutant gene product undergoes conformational maturation and is deployed to its site of action.
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Sifers, R. Manipulating proteostasis. Nat Chem Biol 6, 400–401 (2010). https://doi.org/10.1038/nchembio.374
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DOI: https://doi.org/10.1038/nchembio.374
