Clearing Conformational Disease

See allHide authors and affiliations

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

You are currently viewing the summary.

View Full Text

Log in to view the full text

Log in through your institution

Log in through your institution


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).