A study appearing April 14 in the journal Neuron suggests there may be a new way to change the damaging course of Huntington disease.
University of California, Irvine neurobiologists Leslie Thompson and Joseph Ochaba and their colleagues from UCI and from Children’s Hospital of Philadelphia have shown that reducing the aberrant accumulation of a particular form of the mutant Huntington protein corresponds to improvement in symptoms and neuroinflammation in HD mice.
They targeted and modulated levels of PIAS1 – a protein implicated in cancer and other diseases – which they found led to the reduction of the mutant Huntington protein. The work suggests that changing levels of the PIAS1 protein and targeting this pathway could have a benefit to disease. There are no current treatments for HD, although Thompson’s ongoing work with stem cell-based therapies are showing promise.