Technologies

The development of genome editing tools has revolutionized the way we think and deal with genetics. The use of Cas9 or its variants allows modifications of specific sites in the human genome by inducing deletions and insertions in a more or less controlled way. In recent years, a new class of tools for genome editing has emerged: the base editors (BE), which result from the fusion of a modified Cas9, which serves to direct the BE to the target, and an active deaminase acting on the DNA, which mediates the C> T or A> G editing.

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.