Nanoneedles deliver a base editor to correct a skin-disease mutation
In Advanced Materials, nanoneedle delivery of an adenine base editor corrects a COL7A1 variant in patient cells with recessive dystrophic epidermolysis bullosa.
Base editing can precisely correct single-letter mutations, but delivering base editors efficiently remains a bottleneck. Salman Mustfa, Marija Dimitrievska, Cong Wang and colleagues show that nanoneedles deliver an adenine base editor that corrects a pathogenic COL7A1 variant in primary RDEB fibroblasts with 96.5% efficiency, without inducing off-target variants.
Mustfa SA, Dimitrievska M, Wang C, … Chiappini C. Advanced Materials (2025). doi:10.1002/adma.202414728