内体
细胞内
免疫原性
基因传递
胆固醇
信使核糖核酸
遗传增强
抗原
细胞生物学
化学
生物化学
免疫学
生物
基因
作者
Okhee Jung,Hye‐youn Jung,Lê Thị Thanh Thủy,Minyoung Choi,Seong-Yeon Kim,Hae‐Geun Jeon,Jihyun Yang,Seok‐Min Kim,Tae‐Don Kim,Eunjung Lee,Yoonkyung Kim,Joon Sig Choi
标识
DOI:10.1002/adhm.202303857
摘要
Recently, mRNA-based therapeutics, including vaccines, have gained significant attention in the field of gene therapy for treating various diseases. Among the various mRNA delivery vehicles, lipid nanoparticles (LNPs) have emerged as promising vehicles for packaging and delivering mRNA with low immunogenicity. However, while mRNA delivery has several advantages, the delivery efficiency and stability of LNPs remain challenging for mRNA therapy. In this study, an ionizable helper cholesterol analog, 3β[L-histidinamide-carbamoyl] cholesterol (Hchol) lipid is developed and incorporated into LNPs instead of cholesterol to enhance the LNP potency. The pK
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