In the perspective of developing new therapeutic approaches for these diseases, our laboratory is focused on the latest techniques of cellular reprogramming. These techniques allow the obtainment of induced pluripotent stem cells (iPSCs) from human patients and healthy subjects. These patient-specific cells are useful as model of disease, cell sources for cell-mediated therapy and are strategies for the development of new therapies.
Our laboratory has demonstrated that the transplantation of neural stem cells (NSCs) and motor neuron precursors exert a positive effect on the disease phenotype in mouse models of motor neuron disease, (Corti et al., 2006-2013), supporting the potential cell-mediated therapeutic approach for these diseases.
Moreover, the research team is focused also on another promising therapeutic approach for these neurodegenerative disorders: the use of antisense oligonucleotides (ASO) or morpholino (MO) aim to modify the RNA in order to correct the defect of the disease at the molecular level. They are also ongoing gene therapy approaches based on the traditional introduction of a healthy copy of the gene using vectors, for the autosomal recessive disorders.
This research is the combined contribution of a collaboration between medical doctors, researchers, trainees, students of Medicine, Biology, and Biotechnology, and researchers of national and international laboratories. The teaching activity and the seminars enable the dissemination of the knowledge acquired by our group at national and international level.