Gene editing to correct inherited liver disorders has promise for future therapeutic intervention, but lack of effective and safe delivery of the gene-editing machinery to hepatocytes complicates its clinical application. Two studies now report efficient delivery to the liver of non-human primates, providing proof of concept for novel treatment of inherited hypercholesterolaemia.

中文翻译：

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成人肝脏中的治疗碱基编辑

用于纠正遗传性肝病的基因编辑有望用于未来的治疗干预，但缺乏将基因编辑机制有效和安全地输送到肝细胞使其临床应用复杂化。现在有两项研究报告了非人类灵长类动物肝脏的有效递送，为遗传性高胆固醇血症的新疗法提供了概念证明。