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Therapeutic approaches to protein-misfolding diseases

Nature volume 426, pages 905–909 (2003)Cite this article

Abstract

Several sporadic and genetic diseases are caused by protein misfolding. These include cystic fibrosis and other devastating diseases of childhood as well as Alzheimer's, Parkinson's and other debilitating maladies of the elderly. A unified view of the molecular and cellular pathogenesis of these conditions has led to the search for chemical chaperones that can slow, arrest or revert disease progression. Molecules are now emerging that link our biophysical insights with our therapeutic aspirations.

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Figure 1: Folding of mutant proteins destined to be lysosomal enzymes.

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Author information

Authors and Affiliations

  1. Department of Cellular and Molecular Pharmacology, University of California at San Francisco, Genentech Hall, 600 16th Street N472J, San Francisco, 94107, California, USA

    Fred E. Cohen

  2. Department of Chemistry and the Skaggs Institute of Chemical Biology, The Scripps Research Institute, 10550 North Torrey Pines Rd, BCC 265, La Jolla, 92037, California, USA

    Jeffery W. Kelly

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  1. Fred E. Cohen
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Cohen, F., Kelly, J. Therapeutic approaches to protein-misfolding diseases. Nature 426, 905–909 (2003). https://doi.org/10.1038/nature02265

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