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Dual-functional poly(disulfide) enables “Once-and-for-all” atherosclerosis therapy via precision hepatocyte base editing
A dual-action molecule enables precise liver cell gene editing for one-time atherosclerosis treatment
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Abstract
A single administration of a dual-functional poly(disulfide) resulted in durable editing of the ANGPTL-3 gene in a mouse model of atherosclerosis.
- The poly(disulfide) facilitates hepatocyte-specific targeting through galactose ligands that bind to asialoglycoprotein receptors.
- Direct cytosolic delivery of adenine base editors is achieved by bypassing endosomal entrapment via thiol-disulfide exchange.
- One-dose treatment led to sustained reductions in low-density lipoprotein cholesterol levels.
- Significant attenuation of plaque formation was observed following the single-dose treatment.
- This method represents the first successful application of base editing for atherosclerosis prevention and treatment.
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