GalNAc-Lipid Nanoparticles Enable Non-LDLR Dependent Hepatic Delivery of a CRISPR Base Editing Therapy
3 Articles
3 Articles
GalNAc-Lipid nanoparticles enable non-LDLR dependent hepatic delivery of a CRISPR base editing therapy
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…
Advanced delivery systems for gene editing: A comprehensive review from the GenE-HumDi COST Action Working Group
In the past decade, precise targeting through genome editing has emerged as a promising alternative to traditional therapeutic approaches. Genome edit…
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