Reacción a "Harmful mutations in human mitochondrial DNA corrected through gene editing"

Gene therapy for mitochondrial diseases, which are maternally inherited, is difficult because the entry of nucleic acids into the mitochondria is highly restricted. This means that these diseases, which involve mutations in mitochondrial DNA, have lagged behind in the design of therapies, as the conventional CRISPR gene editing strategy is not possible because the guide RNA cannot enter the mitochondria. The interest of this research lies in the fact that the researchers have used an alternative strategy that requires only the entry of proteins into the mitochondria. They have used base editors derived from a bacterial toxin fused to TALEN proteins that direct the editors to specific sequences of mitochondrial DNA. With this strategy, they have managed to generate a model of mitochondrial disease in liver organoids and correct, to a certain extent, fibroblasts from patients affected by a mitochondrial disease. It is also interesting that they have found that the delivery of the necessary components for base editing is more efficient if this machinery is transferred using modified RNA wrapped in lipid particles, in a manner very similar to how COVID-19 RNA vaccines were delivered.