Click Chemistry for Future Gene Therapies to Benefit Citizens, Researchers and Industry

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Gene therapy is expected to play a key role in next-generation medicine by correcting the underlying genetic causes of disease, thereby facilitating personalised medicine. Current gene therapy methods possess undesirable side effects, including insertional mutagenesis, toxicity, low efficiency and off-target cutting. Questions also remain regarding the optimal methods for delivering nucleases into cells and tissues. These limitations will be addressed through the original and innovative approach of the ClickGene network. ClickGene’s research objectives are to train 14 ESRs in the field of biomaterials development with specific focus on, i.) site-selective genetic engineering, ii.) liposomal nanoparticle drug delivery, and iii.) optogenetic diagnostic probes for epigenetic base detection. Most of our academic partners are key members of COST Action CM1201: Biomimetic Radical Chemistry and all have outstanding international reputations as scientific leaders in their field. This COST Action will play a vital role in both networking and training elements of ClickGene, and in combination with transferable skills training, intersectorial and cross-disciplinary secondment, and industry targeted workshops, the education of biomaterials chemists with expertise in click-chemistry and cutting edge application areas of gene therapy, nanotechnology and biological diagnostics will be achieved. The training provided will match the skills required by next-generation research leaders in biopharmaceutical, nanotechnology, biodiagnostic and other industries. Allied to academic strength in this network, ClickGene features highlycompetitive industry partners with expertise in commercialisation of nucleic acid click-chemistry (baseclick, ATDBio) and liposomal and lipidome technology (LipiNutraGen). Thus, synergy between both commercial and academic partners will enable ESRs to develop gene-targeted technology within a highly productive, engaging, and exciting training network.