Modelling Huntington's disease with human iPSC-derived neurons

Huntington’s disease is a progressive neurodegenerative disorder caused by an abnormal expansion of CAG repeats within the Huntingtin gene. Treating this disease remains a substantial challenge, in part because researchers lack reliable cell models of Huntington’s disease. Patient-derived iPSC-derived cells are difficult to obtain and require complex differentiation protocols, which limit the scalability and reproducibility of any Huntington’s disease model. Additionally, the engineering of the Huntington's disease genotype has proven a significant challenge to the generation of a robust Huntington’s disease cell line.

We have developed ioDisease Model Cells with the aim of solving these challenges. Drug discovery experts can access defined, consistent, and scalable human iPSC-derived neurons with abnormal CAG repeat expansions in the HTT gene. Microelectrode array analysis demonstrates delayed neuronal network formation and decreased spontaneous activity in the HTT 50CAG/WT neurons relative to the genetically matched control. The cells are suitable for use in phenotypic screening assays, helping improve the translatability of in vitro Huntington’s disease research for the development of new therapies.

Discover ioDisease Model Cells for Huntington’s disease research below.