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Small structural changes in siloxane-based lipidoids improve tissue-specific mRNA delivery
Nature Nanotechnology ( IF 38.1 ) Pub Date : 2024-10-28 , DOI: 10.1038/s41565-024-01748-5


From a single library of siloxane-based lipidoids, siloxane-incorporated lipid nanoparticles (SiLNPs) involving minor alterations in lipid chemistry yield tissue-specific mRNA delivery to the liver, lung, or spleen. Upon enhanced intracellular delivery, these SiLNPs show clinical promise for protein replacement therapies, regenerative medicine, and CRISPR–Cas-based gene editing applications.

中文翻译:


基于硅氧烷的类脂质的微小结构变化改善了组织特异性 mRNA 递送



硅氧烷掺入脂质纳米颗粒 (SiLNP) 涉及脂质化学的微小改变,从单个基于硅氧烷的脂质类化合物文库中,产生组织特异性 mRNA 递送至肝脏、肺或脾脏。在增强细胞内递送后,这些 SiLNP 在蛋白质替代疗法、再生医学和基于 CRISPR-Cas 的基因编辑应用中显示出临床前景。
更新日期:2024-10-28
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