Neuroscience

Stem cells and neurogenesis

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Group leader

Vania Broccoli

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Nothing in life is to be feared, it is only to be understood. Now is the time to understand more, so that we may fear less. 

Marie Curie

Our lab has a strong interest in developing novel technologies in stem cells, genetic cell reprogramming and CRISPR/Cas9 gene editing for better modeling and treating neurological disorders. Patient’s derived iPS cells (iPSCs) offer a superior cellular model to recapitulate the key pathophysiological defects underlying the disease. In addition, CRISPR/Cas9 gene-editing provides a fast and efficient system to prove the direct association between a gene mutation and a specific cellular trait.  CRISPR/Cas9 gene editing is a crucial tool in the lab to generate isogenic control iPSCs or to introduce targeted gene mutations. Lately, we have conceived and validated new approaches for correcting mutated genes or modulating their expression by CRISPR technology in vitro and in vivo. For instance, we are using targeted gene boosting to establish novel translational approaches to treat Dravet syndrome and Friedreich’s ataxia. To vehiculate these tools in the brain and set up strategies of in vivo gene-therapy, this lab is producing new variants of adeno-associated viruses (AAV) that combined high targeting efficiency, tissue spreading and safety. Directed evolution screenings of AAV peptide display libraries or tailored modifications of the viral capsids are exploited to identify engineered variants able to acquire new neurotropic abilities including crossing of biological barriers, maximal spreading in the tissue and minimal immune reactivity. Finally, we are exploring the molecular processes that modulate the intricate interplay between neuroinflammation, adaptive immune response and neurodegeneration in new experimental models of Parkinson’s disease recently developed in our lab. This new knowledge will enable us to conceive new therapeutic approaches aiming to dampen the detrimental immune reaction which contributes to the disease progression in Parkinson’s disease.