Modify ALS (ModALS): Integration of OMICs and digital biology in mouse and human motor neuron models for fast-track therapeutic innovation

• Applicant: Professor Dr. Jochen Weishaupt
• Subject Area: Molecular and Cellular Neurology and Neuropathology ; Experimental Models for the Understanding of Nervous System Diseases
• Term: since 2023
• Project identifier: Deutsche Forschungsgemeinschaft (DFG) - Project number 530064068

ModALS aims to develop a new and comprehensive platform for identifying therapeutic molecules for Amyotrophic Lateral Sclerosis (ALS). The strategy is based on high-end machine learning-assisted data analysis of patient-derived human motor neurons (MNs) and animal models. The main goals of this proposal can be summarized as follows: • Development of highly predictive translational models capable of capturing the biological complexity of both sporadic and familial ALS. • New mechanistic insights into the early and asymptomatic phases of ALS when compensatory strategies are active and can be boosted by therapeutic molecules. • Deciphering age- and context-dependent genes, biological processes and molecular pathways that contribute to ALS onset and progression and play context-specific roles in juvenile and aged conditions. • Identification of transcriptomic and phenotypic profiles that can guide the discovery of new harmonized screenable markers and read-outs for therapeutic intervention.

• DFG Programme: Research Grants
• International Connection: France
• Co-Investigator: Dr. Rosanna Parlato
• Cooperation Partners: Dr. Christian Neri; Dr. Peter Sommer