The Niemann-Pick C1 Inhibitor NP3.47 Enhances Gene Silencing Potency of Lipid Nanoparticles Containing siRNA

The therapeutic applications of lipid nanoparticle (LNP) formulations of small interfering RNA (siRNA), are hampered by inefficient delivery of encapsulated siRNA to the cytoplasm following endocytosis. Recent work has shown that up to 70% of endocytosed LNP-siRNA particles are recycled to the extra...

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Detalhes bibliográficos
Publicado no:Mol Ther
Main Authors: Wang, Haitang, Tam, Yuen Yi C, Chen, Sam, Zaifman, Josh, van der Meel, Roy, Ciufolini, Marco A, Cullis, Pieter R
Formato: Artigo
Idioma:Inglês
Publicado em: Nature Publishing Group 2016
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Original Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5167785/
https://ncbi.nlm.nih.gov/pubmed/27633442
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2016.179
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