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The limited packaging capacity of adeno-associated viruses (AAVs) challenges their use as DNA base editor delivery vehicles. Davis et al. have constructed AAVs with size-optimized genomes incorporating small, highly active variants of the adenine base editor (ABE) 8e. In mice, injection of the single-AAV-encoded ABEs supported base editing efficiencies of 66%, 33% and 22% in liver, heart and muscle, respectively, outperforming dual-AAV approaches. Efficient knockdown of circulating human PCSK9 and mouse PCSK9 and ANGPTL3, along with reductions of plasma cholesterol and triglycerides was achieved. The ABEs exhibited broad protospacer adjacent motif compatibility, for editing ~82% of adenines in the human genome.
Davis, J. R. et al. Efficient in vivo base editing via single adeno-associated viruses with size-optimized genomes encoding compact adenine base editors. Nat. Biomed. Eng.https://doi.org/10.1038/s41551-022-00911-4 (2022)