Gavin D. Kenny1, 2, Alison Bienemann3, Katharina Welser4, Frederick Campbell4, Aristides D. Tagalakis1, Mauro Botta5, Alethea B. Tabor4, Ed White3, Mark F. Lythgoe2, Stephen L. Hart1
1Molecular Immunology Unit ICH, UCL, London, United Kingdom; 2Centre for Advanced Biomedical Imaging, UCL, London, United Kingdom; 3Functional Neurosurgery Group, University of Bristol, Bristol, United Kingdom; 4Department of Chemistry, UCL, London, United Kingdom; 5Dipartimento di Scienze dell 'Ambiente e della Vita, Universit del Piemonte Orientale "Amedeo Avogadro", Alessandria, Italy
Genetic therapies offer great promise for the development of new therapeutics in the CNS. One of the major obstacles to overcome is the inability to effectively bypass the BBB and deliver the therapeutic nucleic acids to the affected region. Nanoparticles are extensively used as delivery vectors for genes due to their biocompatibility and the protection they afford the gene and if administered using convection enhanced delivery the BBB can be circumvented. Here we have investigated the use of anionic nanoparticles, that are MR sensitive, contain labels for fluorescence microscopy/histology, in combination with a targeting peptide to mediate the functional delivery.