UC Riverside Scientists Breakthrough: New Hope in Treating Huntington’s and Other Neurodegenerative Diseases

A groundbreaking discovery by scientists at the University of California, Riverside, offers new hope in the treatment of Huntington’s disease, a currently incurable condition marked by involuntary movements and dementia. The research team, led by distinguished UCR professor of chemistry Yinsheng Wang, has successfully slowed the progression of the disease in model organisms such as flies and worms, a significant stride towards developing human treatments.

The key to these advancements lies in understanding how genetic information in cells transitions from DNA to RNA, and then into proteins. Huntington's disease is associated with the expansion of three nucleotides, cytosine-adenine-guanine (CAG), in DNA. This expansion leads to longer RNA repeats, which undergo methylation, a type of chemical modification. Wang and his team discovered that this methylation process converts essential cellular proteins into waste, exacerbating the disease.

This breakthrough, detailed in a recent Nature journal article, is crucial as it mirrors observations in brain tissues of patients with Huntington’s disease, ALS, and frontotemporal dementia. The researchers managed to significantly reduce disease progression and extend lifespan in model organisms by introducing a protein that removes RNA methylation.

Currently, there is no cure for Huntington’s disease, and treatment options are limited to managing symptoms. However, this discovery opens the door to developing effective therapies. The research team, including professors Weifeng Gu at UCR, X. William Yang at UCLA, and Nancy M. Bonini at the University of Pennsylvania, is now exploring small molecules that can inhibit methylation, potentially forming the basis for a Huntington’s therapy.

Moreover, since similar RNA repeats are present in other neurodegenerative diseases like ALS and certain types of spinocerebellar ataxia, this discovery holds promise for treating these conditions as well. While acknowledging that multiple mechanisms contribute to Huntington’s, Wang is optimistic that targeting methylation can improve the lives of those suffering from these debilitating diseases.

This pioneering study not only sheds light on the complex mechanisms underlying Huntington’s disease but also represents a beacon of hope for those affected by this and potentially other neurodegenerative disorders.