In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates.

Nature64.80
Volume: 593, Issue: 7859, Pages: 429 - 434
Published: May 1, 2021
Abstract
Gene-editing technologies, which include the CRISPR–Cas nucleases1–3 and CRISPR base editors4,5, have the potential to permanently modify disease-causing genes in patients6. The demonstration of durable editing in target organs of nonhuman primates is a key step before in vivo administration of gene editors to patients in clinical trials. Here we demonstrate that CRISPR base editors that are delivered in vivo using lipid nanoparticles can...
Paper Details
Title
In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates.
DOI
Published Date
May 1, 2021
Journal
Volume
593
Issue
7859
Pages
429 - 434
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