Huntington's Disease (HD) Model based In Vitro Assay Services

Huntington's Disease (HD) is a devastating inherited neurodegenerative disorder stemming from mutations within the HTT gene. This gene, located on chromosome 4, provides the blueprint for the huntingtin protein, which is normally expressed in the brain. However, when the CAG repeat sequence within the HTT gene abnormally expands, it sets off a cascade of events leading to the abnormal accumulation of mutant huntingtin protein. This buildup ultimately triggers the progressive degeneration and death of nerve cells, resulting in the characteristic motor, cognitive, and psychiatric symptoms of HD. Our services encompass comprehensive in vitro, in vivo, and ex vivo assays to meet your specific needs. Furthermore, our services include personalized customization options, which can accelerate the development of neuropharmacology validation experiments, a critical component of HD research.

Available In Vitro Models

Fig.1 Characterizing GABAergic medium spiny-like neurons derived from human pluripotent stem cells (PSCs).¹

Cell models	Details
iPSC-derived Neuronal Models	Revolutionize your HD research with our cutting-edge 3D culture system, optimized for iPSC-derived striatal neurons. This innovative platform allows you to create a physiologically relevant environment, mimicking the complex cellular interactions of the striatum. By combining our system with iPSC-derived neurons differentiated from HD patients, you can model the CAG repeat expansion phenotype in vitro, unlocking new insights into disease mechanisms and accelerating drug discovery. Our system offers unparalleled control and reproducibility, ensuring reliable data and advancing your HD research.
Reprogrammed Striatal Neuron Model	Through directly reprogramming skin fibroblasts from HD patients into striatal neurons, the patient's original epigenetic information is retained. This model can reproduce the aggregation of mutant huntingtin protein (mHTT) and independently evaluate the progression of neuropathology, making it suitable for accelerating drug discovery and advancing HD research.
Coronal Tissue Organ Slice Model	A new HD model, using mechanical transection of GABAergic neuronal pathways (e.g., substantia nigra pars reticulata) instead of neurotoxins, preserves brain region organization and synaptic circuits. This cost-effective and simple model is ideal for studying neural activity and drug screening.
Cell Line Models	HT22 mouse hippocampal neurons: simulate neuronal damage by overexpressing mutant huntingtin protein for drug screening and neuroprotective mechanism research. Other cell lines (such as PC-12, SH-SY5Y, N2a): study protein aggregation and cytotoxicity by expressing mHTT fragments with different CAG repeat lengths.

Available HD In Vitro Services

Our CRO platform is committed to accelerating the development of HD treatments through relentless technological innovation and unparalleled service. We empower our clients with the efficiency and precision they need to make a difference.

mHTT Aggregation/Lowering Assay

HTT Splicing Assay

Ready to accelerate your HD research? Our expert team and cutting-edge in vitro services can help you unlock new discoveries and advance the development of effective therapies. Contact us today to explore how our comprehensive platform can support your research goals.

Reference

1. Nekrasov, Evgeny D., et al. "Manifestation of Huntington's disease pathology in human induced pluripotent stem cell-derived neurons." Molecular neurodegeneration 11 (2016): 1-15. Distributed under Open Access license CC BY 4.0, without modification.