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Using Patient-Specific Cells for Disease Modelling
Authors: Jessica Tilman, Axol Bioscience Ltd | Last updated: August 2023
Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disorder caused by the degeneration of motor neurons, and ultimately results in death. Despite the fatal nature of this disease, there is no current cure or effective treatment available. The clear overlap between ALS and Frontotemporal Dementia (FTD) has led to the belief that it is not only disruption in motor neurons which drives ALS symptoms, but other cell types such as microglia, the main immune cell found in the brain, may also play a role.
iPSC-derived cells provide a valuable tool for disease modelling, with the ability to use cells directly from affected patients, as well as the potential to drive these cells towards multiple lineages. In this study, axoCells™ motor neurons and microglia from the same healthy and ALS axoLines™ iPSCs were shown to exhibit disease phenotypes which can be quantified and used for future drug discovery purposes.
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