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GalNAc-Lipid Nanoparticles Enable Non-LDLR Dependent Hepatic Delivery of a CRISPR Base Editing Therapy

Summary by Nature
Lipid nanoparticles have demonstrated utility in hepatic delivery of a range of therapeutic modalities and typically deliver their cargo via low-density lipoprotein receptor-mediated endocytosis. For patients lacking sufficient low-density lipoprotein receptor activity, such as those with homozygous familial hypercholesterolemia, an alternate strategy is needed. Here we show the use of structure-guided rational design in a series of mouse and no…

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News-Medical.net broke the news in on Wednesday, July 9, 2025.
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