San Raffaele Telethon Institute for Gene Therapy

Gene and neural stem cell therapy for lysosomal storage diseases

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Head of Unit

Angela Gritti

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Our work is focused on evaluating novel gene therapy strategies based on lentiviral-mediated gene transfer to the CNS, either as single treatment or coupled to neural and hematopoietic stem cell transplantation (to target the PNS and periphery) to treat neurodegenerative lysosomal storage disorders (LSDs), rare genetic disease casues by genetic deficiency of specific lysosomal enzymes.

A critical part of our research is devoted to clarify the early pathogenic events and the therapeutic mechanisms of disease correction upon treatments, in order to refine and implement the gene therapy strategies under testing.

Research activity

To address this issue we are exploiting several in vitro models, including patient-specific induced pluripotent stem cells (iPSCs)-based 2D and 3D cultures (organoids). We are also expanding our disease portfolio and platforms, exploring CNS-directed gene editing strategies to address currently untreatable CNS disorders of glial cells.