The Biffi Lab@Unipd

The Biffi Lab@Unipd company information, Employees & Contact Information

The Lab is headed by Prof. Alessandra Biffi, her research has always been aimed at enhancing the efficacy of HSC-based therapeutic approaches for lysosomal storage disorders with severe nervous system involvement. The activity of the group is devoted to generating novel therapeutic paradigms and clinical translation of new platforms based on the use of genetically engineered hematopoietic cells in multiple disease areas: i) inherited neurometabolic diseases of childhood: our group is historically focused on the development of novel treatment approaches for inborn errors of metabolisms affecting lysosomal function, namely lysosomal storage disorders, based on the use of hematopoietic stem and progenitor cells and their myeloid progeny as vehicles for therapeutics in the central nervous system. ii) autoimmune disorders: the use of autologous hematopoietic cell transplantation has been widely explored in the context of randomized multicenter clinical trials as a treatment option for autoimmune diseases, such as multiple sclerosis and type 1 diabetes. iii) pediatric cancer, namely AML and neuroblastoma: collaborative projects have been established within our division combining the local unique expertise in pediatric oncology, advanced diagnostics and target discovery with the HC gene transfer and gene therapy expertise of the Biffi’s group. Our group developed and optimized innovative transplant protocols to improve HSC engraftment in the brain, enhancing and accelerating microglia repopulation in recipient mice. We demonstrated that exclusive direct intra-cerebral ventricular delivery of HSCs into myeloablated mice results in efficient and rapid microglia repopulation independently from HSC engraftment in the bone marrow and hematopoietic reconstitution, increasing the specificity, efficiency, and therapeutic potential of this approach for chronic neurodegenerative diseases.
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