Lipid nanoparticles for targeted siRNA delivery - Going from bench to bedside

Timofei S. Zatsepin, Yuri V. Kotelevtsev, Victor Koteliansky

    Research output: Contribution to journalReview articlepeer-review

    115 Citations (Scopus)

    Abstract

    This review covers the basic aspects of small interfering RNA delivery by lipid nanoparticles (LNPs) and elaborates on the current status of clinical trials for these systems. We briefly describe the roles of all LNP components and possible strategies for their improvement. We also focus on the current clinical trials using LNP-formulated RNA and the possible outcomes for therapy in the near future. Also, we present a critical analysis of selected clinical trials that reveals the common logic behind target selection. We address this review to a wide audience, especially to medical doctors who are interested in the application of RNA interference-based treatment platforms. We anticipate that this review may spark interest in this particular audience and generate new ideas in target selection for the disorders they are dealing with.

    Original languageEnglish
    Pages (from-to)3077-3086
    Number of pages10
    JournalInternational Journal of Nanomedicine
    Volume11
    DOIs
    Publication statusPublished - 5 Jul 2016

    Keywords

    • Clinical trial
    • Lipid nanoparticle
    • MRNA
    • RNA therapeutics
    • SiRNA
    • Targeted delivery

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