Technology transfer - NMH and CM-EsH derived from CEDU of ALS patients
Establish protocols to obtain human motor neuronal (NMH) and skeletal muscle (CM-EsH) cells from urine-derived stem cells (CEDU) of ALS patients. This knowledge will be transferred from UC to USAL and IBSAL, consolidating a cross-border collaboration network in the regions of Central Portugal and Castilla y León (Spain). This activity includes conducting an epidemiological study and optimising processes to differentiate NMH and CM-EsH cells, which are fundamental for advancing towards the creation of the three-dimensional model.
Creation of an innovative 3D model for ALS
Design and optimise a three-dimensional model based on cells derived from ALS patients, using the technological advances and knowledge generated in Activity 1. This model will precisely replicate the mechanisms of disease progression and provide an advanced tool for studying its pathophysiology. In addition, the 3D model will be characterised to ensure its reliability as an experimental platform.
Functional characterisation of NMH and CM-EsH cells
Apply advanced techniques to study the metabolic and mitochondrial functions of NMH and CM-EsH cells obtained from ALS patients. This analysis will identify key alterations in cell function and mitophagy processes, which are critical to understanding the disease. Additionally, the potential of mitochondria-targeted compounds as therapeutic strategies will be evaluated.
Validation of personalised therapies in the 3D model
Test the efficacy of antioxidant compounds targeting mitochondria in the developed 3D model, demonstrating their ability to reverse alterations observed in ALS patient cells. This activity will confirm the potential of the 3D model as a translational tool for developing more effective and personalised therapies.
Communication, visibility, and awareness
Translate the scientific results obtained into language accessible to society and the scientific community. Dissemination activities will raise awareness about ALS, highlight the project's impact, and ensure transparency by publishing results in open access, following open science principles.