Optimisation and transfer of cell protocols
Development and transfer of protocols to obtain human motor neuronal (NMH) and skeletal muscle (CM-EsH) cells from urine-derived stem cells (CEDU) of ALS patients. This initial result will lay the foundation for creating three-dimensional models and strengthen technological cooperation between UC, USAL, and IBSAL
Creation of an advanced 3D model for ALS
Design of an intermediate 3D model as the first step towards creating a complete three-dimensional model, exclusively composed of neuronal and muscle cells from the same patient. This model will serve as a platform for advanced research and foster technological transfer within the cross-border network between Central Portugal and Castilla y León.
Functional analysis of cells and 3D models
Study of NMH and CM-EsH cells from the 3D model to identify significant alterations in mitochondrial function and mitophagy processes, two key parameters in ALS pathophysiology. These analyses will enable the identification of potential therapeutic targets and promising strategies, enhancing research and innovation capabilities in the cross-border region.
Validation of personalised therapies in the 3D model
Demonstration of the reversal of mitochondrial dysfunction and mitophagy defects in ALS patient cells using antioxidants. Validation of the 3D model as an effective tool for testing targeted therapies, highlighting its potential to reverse the ALS phenotype and advance towards personalised treatments for patients.