Vania Broccoli

Stem cells and neurogenesis

Vania Broccoli, Head of Unit

The Broccoli’s research Unit 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.
This group has established numerous strategies for direct cell reprogramming to generate induced neuronal and glial cells for accelerating cellular modeling of human disorders. Moreover, they have established iPSCs from patients suffering from various diseases including Dravet, Rett and ASD syndromes, NBIA and Parkinson’s disease. CRISPR/Cas9 gene editing is a crucial tool in the lab to generate isogenic control iPSCs or to introduce targeted gene mutations. Lately, they have conceived and validated new approaches for correcting mutated genes or modulating their expression by CRISPR technology in vitro and in vivo. 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.