基因组编辑在血脂异常中的治疗应用。
Therapeutic application of genome editing in dyslipidemia.
作者信息
Whittaker Madelynn N, Musunuru Kiran
机构信息
Department of Bioengineering, School of Engineering and Applied Science.
Cardiovascular Institute, Department of Medicine, and Department of Genetics, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA.
出版信息
Curr Opin Lipidol. 2022 Apr 1;33(2):133-138. doi: 10.1097/MOL.0000000000000805.
PURPOSE OF REVIEW
To summarize recent advances with respect to the use of genome editing to modify blood lipid levels in vivo.
RECENT FINDINGS
Genome-editing technologies have been successfully used to target the PCSK9 gene in the livers of nonhuman primates and significantly reduce blood LDL cholesterol levels.
SUMMARY
Multiple proof-of-concept nonhuman primate studies raise the prospect of genome editing empowering 'one-and-done' therapies for the treatment of dyslipidemic patients.
综述目的
总结近期在体内使用基因组编辑来改变血脂水平方面的进展。
最新发现
基因组编辑技术已成功用于靶向非人类灵长类动物肝脏中的前蛋白转化酶枯草溶菌素9（PCSK9）基因，并显著降低血液中低密度脂蛋白胆固醇水平。
总结
多项概念验证性非人类灵长类动物研究提出了基因组编辑为血脂异常患者提供“一次治疗，终身受益”疗法的前景。
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