Clearing Conformational Disease

摘要

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.