Researchers at UCLA demonstrated a lipid nanoparticle‑based gene‑editing platform capable of precisely inserting a full‑length healthy CFTR gene into human airway cells, a step toward a universal gene therapy for cystic fibrosis. The team showed targeted insertion in primary airway cells and preservation of physiological expression patterns. The approach uses engineered LNPs to deliver editing components without viral vectors, potentially overcoming size limits that have constrained prior gene‑replacement strategies. The result advances non‑viral gene correction methods applicable to disorders requiring full‑gene restoration, though in‑vivo delivery, durability and safety remain to be demonstrated in preclinical models.
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