Abstract
Base editing, the introduction of a single base correction in genomic DNA, has the potential to treat genetic skin diseases such as recessive dystrophic epidermolysis bullosa (RDEB), which is characterized by mutations in the COL7A1 gene and type VII collagen (C7) deficiency. Hitherto, base editing has been harnessed to correct disease-causing mutations but a reproducible platform for efficient gene correction and a safe approach for clinical translation remain elusive. Adenine base editors (ABEs) convert A-T base pairs to G-C base pairs without requiring double-stranded DNA breaks or donor DNA templates.
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