Mutant-Huntingtin Molecular Pathways Elucidate New Targets for Drug Repurposing

The spectrum of neurodegenerative diseases known today is quite extensive. The complexities of their research and treatment lie not only in their diversity. Even many years of struggle and narrowly focused research on common pathologies such as Alzheimer's, Parkinson's, and other brain diseases have not brought cures for these illnesses. What can be said about orphan diseases? In particular, Huntington's disease (HD), despite affecting a smaller part of the human population, still attracts many researchers. This disorder is known to result from a mutation in the HTT gene, but having this information still does not simplify the task of drug development and studying the mechanisms of disease progression. Nonetheless, the data accumulated over the years and their analysis provide a good basis for further research. Here, we review studies devoted to understanding the mechanisms of HD. We analyze genes and molecular pathways involved in HD pathogenesis to describe the action of repurposed drugs and try to find new therapeutic targets.

Automated summary

The spectrum of neurodegenerative diseases is extensive, and their research and treatment are complex and diverse. Despite years of research on common pathologies, like Alzheimer's and Parkinson's, cures have not been found. However, Huntington's disease, although affecting a smaller population, still attracts researchers. Understanding the mechanisms of this orphan disease is challenging, but accumulated data provide a foundation for further research.