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Gene therapy represents nowadays one of the most challenging promises for the cure of cancer, rare, and severe diseases. The speed of the steps toward it strongly depends on the efficacy and reliability of the molecular tools involved in the manipulation (isolation, reconfiguration, multiplication) and vehiculation (native state preservation, dense packaging, targeted and “stealth” delivery, local protection) of the nucleic acids.
The main objective of TM-Vector project is to develop a highly reproducible macromolecular edifice capable to function as a cooperative carrier for nucleic acids, characterized by the ability to be post-decorated with biochemical and / or pharmacologic active molecules, by virtue of dynamic processes of selective affinity of host-guest type. The particular architecture of the carrying vector will offer three unique functional properties, representing our original contribution to the practice of nucleic acid vectorization: (i) the ability to target various cell receptors; (ii) the possibility to concomitantly vectorize several types of active molecules (e.g. nucleic acid molecules and pro-drug molecules); (iii) the capacity to selectively attach molecules having neat different volumes and conformations.
TM-Vector project could have societal impact, contributing to the improvement of the quality of life of sufferers. The applied research directions could also contribute to the technology of gene and drugs carriers produced at biopharmaceutical scale.