Loading
Gene Editing · Journal article
Biomaterials · May 23, 2026
Early or partial results. Treat as a signal, not a conclusion.
This is a preclinical proof-of-concept study describing a dual-functional polymer that delivers adenine base editors to hepatocytes in a mouse atherosclerosis model, achieving durable ANGPTL-3 editing and reduced LDL cholesterol after single administration. The work is early-stage, lacks quantified efficacy metrics, and has not been tested in humans or compared to standard-of-care therapies.
Preclinical study (mouse model). Mouse model of atherosclerosis; hepatocytes as target tissue in vivo. Intervention: Single-dose dual-functional poly(disulfide) delivering adenine base editors targeting ANGPTL-3 gene in hepatocytes.
Dual-functional poly(disulfide) achieves hepatocyte-specific targeting via galactose ligands and ASGPR binding System mediates efficient ABE delivery via thiol-disulfide exchange, bypassing endosomal entrapment Single-dose treatment produced sustained reductions in low-density lipoprotein cholesterol and attenuated plaque formation in mouse atherosclerosis model
Safety was not reported in the material analysed. Check the source before drawing any conclusion about harm.
This preclinical result suggests a potential therapeutic strategy for atherosclerosis via one-time base editing, but substantial further development—including safety studies, efficacy quantification, and eventual human translation—would be required before clinical use can be considered.
Single-arm preclinical study in mouse atherosclerosis model demonstrating proof-of-concept for a novel delivery approach; lacks human data, comparator arm, and quantified effect sizes.
As stated by the source record.
This preclinical result suggests a potential therapeutic strategy for atherosclerosis via one-time base editing, but substantial further development—including safety studies, efficacy quantification, and eventual human translation—would be required before clinical use can be considered.
Graded across the dimensions that decide whether you should act, each from what the source actually supports. There is no single score, and where a dimension was not assessed it says so.
What is missing. This record has no reported figures. That is a gap in the analysis, not a judgement about the study.
CRISPR-based base editors hold transformative potential for genetic medicine, but their clinical translation is hampered by the need for cell-specific delivery, efficient cytosolic release, and durable activity. Here, we report a dual-functional poly(disulfide) that simultaneously achieves both hepatocyte-specific targeting and direct cytosolic delivery of adenine base editors (ABEs). By displaying galactose ligands for binding to asialoglycoprotein receptors (ASGPRs) on hepatocytes, our polymer enables specific recognition of hepatocytes. Crucially, the poly(disulfide) backbone then facilitates direct cytosolic delivery via thiol-disulfide exchange, bypassing endosomal entrapment. This dual-function system mediates efficient ABEs delivery to hepatocytes, resulting in durable editing of the ANGPTL-3 gene after a single administration. In a mouse model of atherosclerosis, this one-dose treatment produced sustained reductions in low-density lipoprotein cholesterol and significantly attenuated plaque formation. To our knowledge, this represents the first successful application of base editing for the effective prevention and treatment of atherosclerosis with a single-dose. Our work establishes a "once-and-for-all" atherosclerosis treatment that creates a transformative platform for precision genome medicine in atherosclerosis and other metabolic diseases.
Taken from the source record, never inferred. Follow any of these and new work involving them reaches your briefing.