Lipopeptide nanoparticles for potent and selective siRNA delivery in rodents and nonhuman primates

siRNA therapeutics have promise for the treatment of a wide range of genetic disorders. Motivated by lipoproteins, we report lipopeptide nanoparticles as potent and selective siRNA carriers with a wide therapeutic index. Lead material cKK-E12 showed potent silencing effects in mice (ED(50) ∼ 0.002 m...

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Main Authors: Dong, Yizhou, Love, Kevin T., Dorkin, J. Robert, Sirirungruang, Sasilada, Zhang, Yunlong, Chen, Delai, Bogorad, Roman L., Yin, Hao, Chen, Yi, Vegas, Arturo J., Alabi, Christopher A., Sahay, Gaurav, Olejnik, Karsten T., Wang, Weiheng, Schroeder, Avi, Lytton-Jean, Abigail K. R., Siegwart, Daniel J., Akinc, Akin, Barnes, Carmen, Barros, Scott A., Carioto, Mary, Fitzgerald, Kevin, Hettinger, Julia, Kumar, Varun, Novobrantseva, Tatiana I., Qin, June, Querbes, William, Koteliansky, Victor, Langer, Robert, Anderson, Daniel G.
Formato: Artigo
Idioma:Inglês
Publicado: National Academy of Sciences 2014
Assuntos:
Physical Sciences
Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC3964096/
https://ncbi.nlm.nih.gov/pubmed/24516150
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.1322937111
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