Human Neuronal Cells Differentiated from Urine-derived Stem Cells as a Platform for Personalized Medicine in Amyotrophic Lateral Sclerosis

Overview

Project Summary

Amyotrophic lateral sclerosis (ALS) disease is a neurodegenerative disease that often appears silently, without showing its full range of symptoms. Patients are often misdiagnosed until the disease is clearly manifested. ALS rapidly evolves forcing patients to confront a harsh reality: face a highly debilitating disease that gradually implies more human and mechanical support, which can quickly lead to death, but in which the mind is still clear until the very end. ALS is characterized by progressive degeneration of both upper and lower motor neurons (motor neurons in the cortex, brainstem and spinal cord), which leads to paralysis and death within 2–5 years after diagnosis. Out of the box research is required to counteract the fact that obtaining brain and muscular tissue is not possible without endangering the patient's health, and that animal models do not fully represent the ALS characteristics. This research proposal aims to create an innovative platform for personalized medicine, based on human neurons differentiated from urine-derived stem cells, isolated from ALS patients' urine. As a proof of concept for personalized medicine, several mitochondria-targeted active antioxidants will be tested.

Main Goals

This interdisciplinary research proposal aims to create an innovative personalized medicine platform for ALS, based on human neuronal cells differentiated from UDSC isolated from ALS patients' urine. The study of these cells will allow us classifying and stratifying specific features of ALS patients. Given that mitochondrial dysfunction contributes to neurodegeneration in both sALS and fALS, we also aim to test several mitochondria-targeted active molecules in HCN obtained from different ALS patients, as a proof of concept for personalized medicine.

Project Details