bit.bio's new Huntington's disease human cell model, for in vitro research and drug discovery

The life sciences industry is challenged by a lack of reliable, reproducible cell models that generate translatable, reflective, data of actual human disease. ioGlutamatergic Neurons HTT^50CAGWTaims to provide a viable solution for HD. CRISPR/Cas9 gene editing has been used to introduce an abnormal expansion of 50 CAG repeats into the first exon of the Huntingtin gene within the wild type ioGlutamatergic Neurons. These highly characterised iPSC-derived glutamatergic neurons accurately represent the disease genotypein vitro. The engineered cells rapidly mature into functional excitatory neurons that consistently form complex neuronal structures and express typical biomarkers in as little as 11 days.

ioGlutamatergic Neurons HTT50CAGWTprovide batch-to-batch consistency at a scale of billions of cells, forming a reproducible model enabled by opti-oxTM precision reprogramming. In addition, bit.bio’s wild type ioGlutamatergic Neurons can be used as a genetically matched control for the ioGlutamatergic Neurons HTT50CAG/WT disease model, offering a physiologically-relevant isogenic pairing with which to ...