Gene expression from embryoid bodies derived from HBG3 HB9 ES cells exposed to serum from a murine Huntington's disease model. Mus musculus

We have established proof of principle for the Indicator Cell Assay Platformé (iCAPé), a broadly applicable tool for blood-based diagnostics that uses specifically-selected, standardized cells as biosensors, relying on their innate ability to integrate and respond to diverse signals present in patients' blood. To develop an assay, indicator cells are exposed in vitro to serum from case or control subjects and their global differential response patterns are used to train reliable, cost-effective disease classifiers based on a small number of features. In a feasibility study, the iCAP detected pre-symptomatic disease in a murine model of amyotrophic lateral sclerosis (ALS) with 94% accuracy (p-Value=3.81E-6) and correctly identified samples from a murine Huntington's disease model as non-carriers of ALS. In a preliminary human disease assay, the iCAP detected early stage Alzheimer's disease with 72% cross-validated accuracy (p-Value=3.10E-3). For both assays, iCAP features were enriched for disease-related genes, supporting the assay's relevance for disease research. Overall design: Embryoid body derived motor neurons from HBG3 HB9 ES cells.