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A clinician-focused summary of the 2026 ACC Scientific Statement on gene editing for cardiovascular disease, highlighting indications, recommendations, safety monitoring, ethical issues, and practical steps for integrating these therapies i
A 6-year-old girl died seven days after receiving a personalised base-editing therapy for a rare genetic disorder in an unregistered Chinese trial, prompting international concern about oversight.
A phase 1, ascending‑dose study of CTX310 (LNP-delivered CRISPR-Cas9 targeting ANGPTL3) in 15 patients produced dose-dependent ANGPTL3 reductions at ≥0.6 mg/kg with few acute safety signals; longer follow‑up and larger trials are required t