Dendrimers as Non-Viral Vectors in Gene Therapy
Gene therapy is currently limited in clinical applications due to the difficulty of delivering genetic material into cells in a safe and efficient manner. Dendrimers are a class of highly branched macromolecular synthetic compounds, which are proposed as polymeric non-viral vectors. Dendrimeric structures combined with one or more nucleic acid molecules (DNA or RNA) are called supramolecular complexes.
Dendrimers are highly branched synthetic macromolecules with repetitive structures. These are characterized by a central core, layers of repeating units and active terminal groups with surface charges. Fluorinated amphiphilic dendrimeric structures are capable of assembling into supramolecular systems of different size and shape. The structure comprises a polyester-based hydrophilic portion. The object of the present invention (TRL 4) are fluorinated amphiphilic dendrimeric structures capable of self-assembling in supramolecular systems of different size and shape. The object structures comprise a fluorinated hydrophobic portion and a polyester-based hydrophilic portion. A further object of the present invention are the supramolecular complexes which comprise at least one of said dendrimeric structures and one or more nucleic acids.
- Gene therapy;
- Treatment of neurodegenerative neurological diseases.
- The dendrimeric structures make it possible to have available a multiplicity of magnetically equivalent fluorine atoms, which allow the use of magnetic resonance imaging;
- The presence of four ethereal bonds in the nucleus accelerates the degradation of the molecule in the environment;
- Superior transfection capacity and reduced cytotoxicity compared to non-viral lipid vectors.