Smart nanocarrier delivery platform for multiplexed CRISPR based Gene Editing

The development of CRISPR/CAS9 technology and related tools for gene editing is a major breakthrough in molecular biology of the current decade. The technology allows very selective modification of genomic material and is able to delete, change or insert genetic material into cells including human cells. The major obstacle to harvest the enormous potential of this very young technology is the delivery of the associated protein (CAS9 or related proteins) and guiding RNA strand(s) to the cytoplasm and the cell nucleus. One key aspect of our delivery approach is the application of our recently developed membrane anchoring strategy for oligonucleotides to allow user friendly multiplexed gene editing with optimized guide RNAs. We aim to provide a platform for targeted and personalized non-viral delivery based on non-toxic lipid nanoparticles and membrane anchored components designed from a chemist’s perspective.