First Human Trial of Base Editing for Cholesterol Control Raises Hope and Safety Concerns

Scientists at Verve Therapeutics in Boston conducted the inaugural human trial of base editing, using the treatment VERVE-101 to deactivate the PCSK9 gene in the liver. The gene regulates ‘bad’ cholesterol, and the one-time injection resulted in a significant LDL reduction of up to 55%. This milestone suggests a potential transformative approach to treat coronary artery disease. However, safety concerns emerged, with two serious adverse events, including a death. The findings, presented at the American Heart Association meeting, indicate both promise and challenges for this ground-breaking gene-editing technique.

Source: https://www.nature.com/articles/d41586-023-03543-z

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