Abstract
Amyotrophic Lateral Sclerosis (ALS) is a fatal neurodegenerative disorder characterized by motor neuron degeneration, leading to progressive muscle weakness and atrophy.
Induced pluripotent stem cell (iPSC) technology enables the conversion of primary samples (blood or fibroblast) from a patient donor into a stem cell and from that stem cell, differentiated into functional cell types, including motor neurons. These motor neurons can be used in in vitro modeling when generated from high quality patient derived iPSCs and differentiated using a small molecule robust process to generate functionally qualified human
iPSC-generated motor neurons.
Axol Bioscience has generated motor neurons from a number of patient and unaffected donor samples. We have previously reported ALS relevant phenotypes using low-throughput electrophysiology. To investigate the utility of iPSC-derived motor neurons in a high-throughput setting, we collaborated with Nanion Technologies. We successfully demonstrated the application of these cells on the SyncroPatch 384 system and report ALS relevant differences between motor neurons from an ALS patient C9orf72 affected line and that of an unaffected donor.
