Motor neuron-derived induced pluripotent stem cells as a drug screening platform for amyotrophic lateral sclerosis

The development of cell culture models that recapitulate the etiology and features of nervous system diseases is central to the discovery of new drugs and their translation onto therapies. Neuronal tissues are inaccessible due to skeletal constraints and the invasiveness of the procedure to obtain them. Thus, the emergence of induced pluripotent stem cell (iPSC) technology offers the opportunity to model different neuronal pathologies. Our focus centers on iPSCs derived from amyotrophic lateral sclerosis (ALS) patients, whose pathology remains in urgent need of new drugs and treatment. In this sense, we aim to revise the process to obtain motor neurons derived iPSCs (iPSC-MNs) from patients with ALS as a drug screening model, review current 3D-models and offer a perspective on bioinformatics as a powerful tool that can aid in the progress of finding new pharmacological treatments.

Automated summary

The development and study of cell culture models that replicate the characteristics of nervous system diseases are crucial for drug discovery and treatment advancement. Induced pluripotent stem cell (iPSC) technology provides an opportunity to create neuronal pathology models due to the difficulty in obtaining neuronal tissues. This article focuses on iPSCs derived from patients with amyotrophic lateral sclerosis (ALS) and explores the process of obtaining motor neuron-derived iPSCs (iPSC-MNs), current 3D models, and the potential of bioinformatics in finding new pharmacological treatments.