NaPro BioTherapeutics Inc reported results of a recent study using its proprietary technology in a novel approach to treating Huntington's disease. These results, presented today at the American Society of Gene Therapy (ASGT) Annual Meeting, highlight the discovery that certain proprietary single-stranded oligonucleotides inhibit aggregation and extend the life of neuronal cells by at least 40 percent in a cell culture model system.
In patients suffering from Huntington's disease, the aggregation of the huntingtin protein and other cellular factors leads to neurotoxicity and cell death; therefore, an ability to block the formation of these aggregates is a promising development. To date there are no effective treatments for this devastating disease.
Dr. Eric Kmiec, a NaPro collaborator and Professor of Biological Sciences at the University of Delaware and Director of Applied Genomics at the Delaware Biotechnology Institute, said, "In studying the use of gene editing to treat Huntington's disease, we were delighted to discover that these short, single-stranded oligonucleotides seem to work in a medicinal way -- as opposed to a genetic way. Therefore, we initiated this study to explore these possibilities."
Huntington's disease is a neurodegenerative disease that is characterized by aggregate formation and cell death in the brain. The disease presents, usually in mid-life, and is characterized by involuntary physical movements, severe emotional disturbance and increasing cognitive decline. In the United States, the prevalence of the disease is about 10 cases per 100,000 people - about 35,000 people in all -- with another 175,000 people genetically at risk.