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

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…

New Software Boosts Genome Editing Accuracy for Genetic Therapies

This innovative software, CRISPRware, optimizes guide RNA design for genome editing, supporting research on genetic disorders and uncharacterized peptides.