Experimental research

About the project

It is under development an innovative approach to gene therapy, using modified liposomes with mucoadesidal properties for DNA delivery to the central nervous system via nasal administration. This strategy was successfully tested on mcs I MPS I, increasing the expression of the Idua gene, reducing the accumulation of mucopolysaccharides in the brain and preventing behavioral changes. The next step includes testing this technology in MPS II (Hunter Syndrome), evaluating repeated administrations and conducting safety and biodistribution studies in non -human primates at the National Primate Center (CENP). With financing from Chiesi Global Rare Diseases, this research can revolutionize the treatment of rare diseases, offering a non -invasive, secure alternative and potential for multiple clinical applications.