OVERVIEW

Huntington’s Disease: Mechanisms and Next - Generation Therapeutics is organized by Neuroscience School of Advanced Studies (NSAS) and will be held from Jun 22 - 26, 2025 at Chetzeron Hotel & Restaurant, Crans Montana, Wallis, Switzerland.

Description:
Huntington’s disease (HD) is a late-onset neurodegenerative disease caused by the inheritance of an expanded CAG tract in exon 1 of the huntingtin gene (HTT). Research over the last several decades in the clinic, in postmortem patient samples, and in a variety of cellular and animal models has revealed that additional somatic expansion of the HTT exon 1 CAG tract in the disease-causing allele (mHTT) is an essential initial step in disease pathogenesis. Although genes involved in somatic CAG expansion have been identified, the influence of this suite of genes on mHTT in different cell types in the human brain has not been determined. Furthermore, the molecular mechanisms by which the expanded mHTT leads to cellular dysfunction and death remain to be determined.

During the Challenge Workshop, attention will be devoted to comparing and contrasting mechanisms identified thus far in humans and model systems to understand HD pathogenesis more fully and to discover commonalities between systems and cell types that are fundamentally important for understanding the disease. The challenge will be to discuss these features of the disease in depth, to identify specific targets that can be targeted for HD therapy, and to discuss strategies and therapeutic entities that might best intervene in HD pathogenesis for the benefit of a broad spectrum of HD patients.