Validation of a Novel Human Research Model for ALS in the Context of Energy Metabolism – MINI.ALS

The aim of the project is to develop and optimize an innovative human in vitro model for studying amyotrophic lateral sclerosis (ALS). The foundation of the project will be reprogrammed cells—fibroblasts obtained from patients. This approach makes it possible to preserve the patient’s epigenetic background and maintain age-related transcriptional signatures, which is crucial in the study of neurodegenerative diseases that progress with age.

Given that ALS patients exhibit disturbances in energy metabolism and that post-mortem analyses reveal altered mitochondrial morphology and excessive glycogen accumulation in spinal cord cells, the project will focus specifically on investigating changes in energy metabolism within patient-derived neuronal cells.