New designs of peptidic and polymeric vectors for protein and gene delivery

Resumo

The expansion ol new therapeutic technologies is limited by the effective, selective and contro/led transport and release of the corresponding biomolecules to !he different tissues, ce/Is and organe/les. Therefore, there is a need to develop conceptually new strategies to transport and deliver proteins or nucleic acids inside cells. In this regard, in this PhD thesis, we present the development of novel ce/1-penetrating peptides and polymeric platforms based on amphipathic scaffolds, which are decorated with functional reactive moieties for enhaced specificity and tunability using dynamic covalent bonds such as alkoxyamines or hydraones. These new vectors were employed in the delivery of proteins, such as lectins and Cas 9 for CRISPA technology, and challenging nucleic acids such as mRNA, following a fully supramolecular strategy.