Porous Silicon Nanoneedles Efficiently Deliver Adenine Base Editor to Correct a Recurrent Pathogenic COL7A1 Variant in Recessive Dystrophic Epidermolysis Bullosa.

ABSTRACT: Base editing, a CRISPR-based genome editing technology, enables precise correction of single-nucleotide variants, promising resolutive treatment for monogenic genetic disorders like recessive dystrophic epidermolysis bullosa (RDEB). However, the application of base editors in cell manufacturing is hindered by inconsistent efficiency and high costs, contributed by suboptimal delivery methods. Nanoneedles have emerged as an effective delivery approach, enabling highly efficient, non-perturbing gene therapies both in vitro and in vivo. Here we demonstrate that nanoneedle delivery of an adenine base editor corrects a heterozygous single-nucleotide pathogenic variant in COL7A1 in primary RDEB fibroblasts in vitro with 96.5% efficiency, without inducing off-target variants. The nanoneedle delive

SUBMITTER: Mustfa SA 

PROVIDER: S-EPMC12038538 | biostudies-literature | 2025 Apr

REPOSITORIES: biostudies-literature