Fig. 4: Hepatic and extra-hepatic editing of ANGPTL3 following treatment with a GalNAc-LNP or standard LNP. | Nature Communications

Fig. 4: Hepatic and extra-hepatic editing of ANGPTL3 following treatment with a GalNAc-LNP or standard LNP.

From: GalNAc-Lipid nanoparticles enable non-LDLR dependent hepatic delivery of a CRISPR base editing therapy

Fig. 4: Hepatic and extra-hepatic editing of ANGPTL3 following treatment with a GalNAc-LNP or standard LNP.The alt text for this image may have been generated using AI.

Targeted amplicon sequencing of the ANGPTL3 target site was performed in tissue samples collected at necropsy following dosing with standard lipid nanoparticles (LNPs) (N = 3) and GalNAc-LNPs (N = 3) at a 2 mg/kg dose. Biodistribution and liver editing of GalNAc-LNPs and standard LNPs is similar in wild type (WT) male cynomolgus NHPs, with little editing seen outside the liver for both LNPs. Each point represents results from an individual animal. LN denotes lymph node. Data are presented as mean values + /- standard deviation. Source data are provided as a Source Data file.

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