Therapeutic base editing in the adult liver

被引：0
|
作者：
Coen C. Paulusma
Piter J. Bosma
机构：
[1] Amsterdam UMC,Tytgat Institute for Liver and Intestinal Research, AGEM
[2] University of Amsterdam,undefined
来源：
Nature Reviews Gastroenterology & Hepatology | 2021年 / 18卷
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D O I：
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学科分类号：
摘要：
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.
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页码：597 / 598
页数：1
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