The research group "Advanced Cellular Models of Neurodegeneration" focuses on the development of cutting-edge cellular models for the study of monogenetic neurodegenerative diseases. Dr. Hauser leads a team dedicated to advancing our understanding of neurodegenerative diseases and identifying novel treatment strategies.
His primary research objectives include:
Development of iPSC-based disease models: Dr. Hauser and his team have generated over 100 induced pluripotent stem cell (iPSC) lines from patients with rare monogenetic neurological disorders. These iPSCs serve as tools for disease modeling, allowing the identification of pathophysiologically relevant cellular phenotypes and contributing to the development of therapeutic strategies.
Preclinical treatment strategies for neurodegenerative diseases: His group has developed innovative preclinical treatment options for monogenetic neurological disorders. This work has led to significant advances in innovative treatment strategies, such as allele-specific targeting strategies using antisense oligonucleotides (ASOs) for diseases like spinocerebellar ataxia type 3 (SCA3) as well as mRNA- or AAV-based approaches for hereditary spastic paraplegia (HSP).
Biomarker discovery for neurodegenerative diseases: His group is actively involved in the discovery and validation of reliable biomarkers for the assessment of pathophysiological processes and therapeutic interventions in slowly progressive neurodegenerative diseases. Our focus includes the identification of metabolic biomarkers using iPSC-derived neural models.
Dr. Stefan Hauser
