期刊
ANGEWANDTE CHEMIE-INTERNATIONAL EDITION
卷 59, 期 35, 页码 14957-14964出版社
WILEY-V C H VERLAG GMBH
DOI: 10.1002/anie.202004994
关键词
genome engineering; lipidoid nanoparticle; mRNA delivery; protein delivery; stimuli responsive
资金
- NCATS NIH HHS [UG3 TR002636] Funding Source: Medline
- NIBIB NIH HHS [R01 EB027170] Funding Source: Medline
Developing safe and efficient delivery systems for therapeutic biomacromolecules is a long-standing challenge. Herein, we report a newly developed combinatorial library of cholesteryl-based disulfide bond-containing biodegradable cationic lipidoid nanoparticles. We have identified a subset of this library which is effective for protein and mRNA delivery in vitro and in vivo. These lipidoids showed comparable transfection efficacies but much lower cytotoxicities compared to the Lpf2k in vitro. In vivo studies in adult mice demonstrated the successful delivery of genome engineering protein and mRNA molecules in the skeletal muscle (via intramuscular injection), lung and spleen (via intravenous injection), and brain (via lateral ventricle infusion).
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