Drug Target For Huntington's Disease?

Scientists from Johns Hopkins School of Medicine in the USA have found a protein in the brains of Huntington's disease sufferers which may provide a target for pharmacological intervention. The protein binds to huntingtin, the product of the Huntington's disease gene, and has been called huntingtin-associated protein-1. The findings were announced at the Society for Neuroscience meeting in San Diego, USA, last week.

The Hopkins researchers feel that the same yeast assay which enabled them to find HAP-1 can be modified to find molecules which prevent it from binding to huntingtin. Despite the fact that they still don't know what huntingtin or HAP-1 actually do, some evidence points to an involvement of HAP-1 in the pathogenesis of the condition. For example, the more mutated the huntingtin protein, the tighter it binds to HAP-1. Also, HAP-1 is only found in areas of neurodegeneration, while the huntingtin gene is ubiquitous.