Prof. Biffi’s main research interest is the development of novel Hematopoietic Stem Cell (HSC)-based gene therapy approaches for inherited and, more recently, acquired disorders affecting the central nervous system (CNS). Engineered HSCs progeny in the CNS of myeloablated transplant recipients represent a vehicle for therapeutic molecule delivery across the blood brain barrier. Prof. Biffi has already proven the efficacy of this innovative approach in animal models and patients affected by lysosomal storage disorders (LSDs) (Biffi, JCI 2004 and 2006; Visigalli, Blood 2010; Biffi, Science 2013; Sessa, Lancet 2016) and her group is currently pursuing research aimed at enhancing the therapeutic potential of this strategy (Capotondo, PNAS 2012; Capotondo, Sci Advances 2017; Peviani, Biomaterial 2019) and broaden its application. This research area, which has been funded by an Advanced ERC grant to Prof. Biffi, is now part of an alliance with industry aimed at the clinical development of the new approach in novel indications. Moreover, the lab hosts research sponsored Altheia Science, a spinoff of the University of Padua funded by Prof. Biffi, dedicated at the development of HSC gene therapy for new autoimmune indications. The laboratory operates in the context of international collaborations such as with Boston Children’s Hospital and Harvard Medical School.