Clearing Conformational Disease

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Science  09 Jul 2010:
Vol. 329, Issue 5988, pp. 154-155
DOI: 10.1126/science.1192681

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Research focused on defining the underlying characteristics of a disease can often lead to new treatment strategies. This is certainly the case for a rapidly expanding group of inherited diseases characterized by the toxic accumulation of a misfolded protein (1). Identifying, and then targeting, the cellular machinery responsible for orchestrating the degradation of these aberrant molecules, rather than correcting the mutations that cause them, represents a paradigm shift from the conventional wisdom associated with gene therapy. On page 229 of this issue, Hidvegi et al. (2) describe how a compound that stimulates autophagy—the process by which a cell destroys its own organelles through lysosomal compartments—can successfully eliminate the toxic effects of misfolded α1-antitrypsin protein in a preclinical mouse model of the associated liver disease (3).