We are a young and dynamic group interested in studying the molecular mechanisms of host-vector interplay in the context of hematopoietic stem cell (HSC) gene therapy of monogenic inflammatory diseases, with particular focus on innate immunity and host responses to transduction (Petrillo et al., Mol Ther., 2015; Kajaste-Rudnitski and Naldini, Hum Gene Ther., 2015). To investigate these aspects, we use high through-put transcriptomic approaches, molecular virology tools, advanced gene delivery platforms, state-of-the-art xenograft in vivo models of human hematopoietic reconstitution as well as relevant in vitro and in vivo models of disease. Our overall goal is to provide molecular insight critical for the development of more stealth and efficient HSC gene therapy strategies.
As part of the SR-TIGET, a world-leading Institute in the fields of gene and cell therapy for the treatment of human genetic diseases, we benefit from a highly competitive, international and scientifically stimulating environment and offer excellent working conditions, including state-of-the-art facilities and infrastructures (Next-Generation Sequencing, Cell Sorting and Imaging, Animal Facilities, GLP laboratories), and access to clinically relevant human samples.
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