MOLECULAR NEUROSCIENCE AND GENE THERAPHY
Research Focus & Expertise
ABOUT THE LAB
The Molecular Neuroscience and Gene Therapy group has three main research aims, ranging from basic to translational research and innovation. First, we aim to develop patient-derived cellular and rodent-based models to study the progression of incurable rare diseases, in particular neurodegenerative disorders. Next, using these novel models, we aim to understand the molecular mechanisms underlying the onset, development, and progression of these disorders, which is crucial to identify novel targets and opportunities for therapeutic intervention. Lastly, we aim to develop advanced disease-modifying therapies, especially those based on gene therapy using viral vectors, which can be translated from the bench to the bedside, creating economic value, and improving the lives of patients suffering from these disorders.