In vivo CRISPR editing to cure brain pathologies

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Dominant-negative mutations produce faulty proteins that act antagonistically to wild type (WT) gene products, thereby decreasing their function. For this reason, classical gene therapy approaches involving exogenous gene delivery are inappropriate, and directly correcting the mutation in the genome represents the best option. CRISPR technology has developed rapidly and inserting DNA into post-mitotic cells such as neurons is now possible, although replacing endogenous sequences remains a challenge. In the lab we are using cutting-edge tools in order to achieve the complete correction of the mutated gene in vivo.