Article

An in vivo CRISPR base editing therapy to inactivate the ANGPTL3 gene: nomination of a development candidate for VERVE-201

2022-10-01

Abstract excerpt

Abstract Background Lowering cumulative exposure to low-density lipoprotein cholesterol (LDL-C) is the primary treatment for patients with homozygous familial hypercholesterolemia (HoFH) and established atherosclerotic cardiovascular disease. Due to access, adherence, and healthcare infrastructure limitations, an important fraction of such patients fail to achieve adequate lowering of LDL-C. Durable inactivation i...

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Literature Corpus work: 3c21c904-20cb-5040-9f0f-99ffc6b88e34
DOI: 10.1093/eurheartj/ehac544.3087

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