Huntington's disease (HD) is an autosomal dominant neurodegenerative disease caused by expansion of a polyglutamine (polyQ) bundles in human huntingtin protein (Htt). However, the molecular mechanisms of Htt proteotoxicity are still unknown, and the muscle phenotypes have not been well studied. At CD BioSciences, we provide drug screening services for Huntington's disease utilizing C. elegans as a model organism to help our clients rapidly identify interventions for HD and decode the underlying mechanisms of drug action.
First described by George Huntington in 1872, Huntington's disease (HD) is characterized by progressive motor deficits, dementia, emotional disturbance, and even neuronal death. It occurs due to a mutation in the huntingtin gene with an abnormal CAG repeat, leading to a variable length N-terminal polyglutamine chain (poly-Q). The polyglutamine expansion destabilizes Htt leading to protein folding and aggregates formation, thereby triggering neurodegeneration (closely connected with age and polyQ tract length) and the disruption of energy metabolism in muscle cells. The investigation of potential HD therapeutic compounds takes more time and cost in mammalian models due to the prolonged time course of this disease. Therefore, a rapid and inexpensive alternative is urgently needed to assess the efficacy of numerous candidate compounds, such as the invertebrate models.
Fig.1 Diagram of Huntington's disease pathogenesis and possible therapeutic targets. (Tabrizi S J, et al., 2022)
As an invertebrate organism, C. elegans has provided an attractive model system due to its unique experimental advantages, its easy genetic manipulation, and high homology of genes and signaling pathways with mammals.
In addition, C. elegans possesses conserved pathways associated with protein misfolding, aggregation, and neurodegeneration that have also been linked to HD.
CD BioSciences has established a state-of-the-art high-throughput drug screening platform utilizing expertise in C. elegans research. Our Huntington's disease drug screening service focuses on constructing C. elegans strains for disease drug screening (which are evaluated by several assays) through proven gene editing techniques, and then utilizing our drug screening platform to perform drug screening.
CD BioSciences is a professional provider of C. elegans model services. We offer a systematic platform that enables rapidly and inexpensively screen potential HD drugs and elucidate pathways of drug action. If you have any projects requiring our service, don't hesitate to contact us. We guarantee our clients the most reliable research services to best match your research goals with more comprehensive data analysis and faster turnaround time.