BioCentury
ARTICLE | Targets & Mechanisms

Turning up the heat on HD

February 18, 2010 8:00 AM UTC

Researchers at Duke University have found small molecules that help cells untangle aggregates of the misfolded protein thought to cause Huntington's disease.1 The molecules are now the lead compounds for Chaperone Therapeutics Inc., a neurology startup focused on therapeutics that stimulate protein folding.

Many neurodegenerative disorders including HD are characterized by intracellular aggregates that are thought to compromise neuronal function and survival. HD pathogenesis involves polyglutamine expansion mutations in huntingtin (HTT) that cause the protein to misfold and aggregate, thus triggering neuron death...