Stem cells and neurogenesis

Stem cells and neurogenesis

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Head of Unit

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. The group has established numerous strategies for direct cell reprogramming to generate induced neuronal and glial cells for accelerating cellular modeling of human disorders. Moreover, we 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, we 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.

Research Activity

  • Modelling cellular pathological defects of Parkinson’s disease with patient-derived iPSCs
  • Developing new approaches of AAV-based gene-therapy to limit dopaminergic neuronal death and dysfunctions in Parkinson’s disease
  • Validating new gene and pharmacological therapeutic approaches for Dravet syndrome based on CRISPR/Cas9 technology
  • Understanding the molecular bases of infantile autism-spectrum disorders caused by mutations in chromatin factors and generation of patients derived iPSC models
  • Technical development of new AAV variants for the gene therapy of brain disorders
  • Validating gene-therapy procedures for incurable neuroinfantile disorders