Technologies

22q11.2DS(DGS) deletion syndrome is a rare and phenotypically variable multiorgan syndrome, currently without any cure. Our aim is to develop a standardized approach to formulate pharmacological products useful for clinical trials direct to prevent some serious clinical manifestations of adolescence and adulthood, such as neuropsychiatric and musculoskeletal diseases, or to eliminate or improve cardiovascular defects during embryonic development.

We have identified the presence of the poorly characterized precursor proNGF-A in human tissues, deposited its coding nucleotide sequence (GenBank MH358394) and demonstrated its neuroprotective and neurotrophic activity in vitro and in vivo. We inserted mutations into the native molecule, identified through computational analysis, which allow proNGF-A production by eukaryotic expression systems, through a method currently validated on a laboratory scale.

Safe, efficient and specific nano-delivery systems are increasingly needed for precision and regenerative medicine and targeted therapies (e.g. anticancer and antimicrobial therapies), as well as for  the cosmetic and nutraceutical sectors’ applications. Despite the appreciable success of synthetic nanovectors, like for example liposomes, their clinical and market application is hampered by some limitations: • large scale production, • low cost production • intrinsic toxicity • limited cellular uptake • limited consumer acceptance.