Base editors (BE) have garnered interest in the field of inherited metabolic diseases for their ability to cause precise point mutations. The novel polymer-lipid nanoparticles can target hepatocytes through ASGPR and complete endosomal escape, and significantly improving the delivery efficiency of BE plasmids.
Gao, D.T. et al. / J. Chin. Pharm. Sci. 2024, 33 (3), 189–200.
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