Researchers demonstrated lipid nanoparticle (LNP) delivery of base editors to correct a rhodopsin RHO c.1030C>T (p.Q344X) mutation implicated in retinitis pigmentosa, achieving functional rescue in preclinical models. The LNP platform offers a non‑viral route to edit somatic retinal cells, reducing vector manufacturing complexity and potentially improving repeat‑dosing flexibility. Base editing is a CRISPR‑derived approach that converts single nucleotides without creating double‑strand breaks, lowering the risk of large indels.
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