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Targeted gene repair is a technique used to correct a mutation at a specific site in an episome or chromosome. It uses synthetic oligonucleotides together with the cell's inherent DNA repair system to direct single base pair changes.
Base editing technology has great potential in treating pathogenic single-nucleotide variations. Using a dual-AAV base editing system, Wu et al. restored visual functions in a mouse model of retinitis pigmentosa.
The fusion of a programmable transcription-activator-like effector (TALE) protein with a nickase, in conjunction with a deaminase, enables efficient and strand-selective DNA base editing. This approach has the potential to advance our understanding and treatment of diseases associated with mutations in the mitochondrial or nuclear genome.
Pre-symptomatic gene editing in preclinical models of hypertrophic cardiomyopathy shows therapeutic promise; clinical studies are now needed to assess safety and efficacy in humans.
The addition of Cas9 target sequences to long single-stranded DNAs, combined with cocktails of small molecules to boost homology-directed repair, leads to marked enhancement of non-viral knock-in efficiency and yield in primary human T cells and other hematopoietic cell types.
Two recent studies published in Nature Biotechnology describe the engineering of circularized guide RNAs, which allow for programmable RNA base editing in vivo, with vastly improved editing efficiency and durability.
