Effects of Local Structural Transformation of Lipid-Like Compounds on Delivery of Messenger RNA


Authors: Li B, Luo X, Deng B, Giancola JB, McComb DW, Schmittgen TD, Dong Y

Journal: Sci Rep

DOI: 10.1038/srep22137

Publication - Abstract

February 26, 2016

Abstract:

Lipid-like nanoparticles (LLNs) have shown great potential for RNA delivery. Lipid-like compounds are key components in LLNs. In this study, we investigated the effects of local structural transformation of lipid-like compounds on delivery of messenger RNA. Our results showed that position change of functional groups on lipid-like compounds can dramatically improve delivery efficiency. We then optimized formulation ratios of TNT-b10 LLNs, a lead material, increasing delivery efficiency over 2-fold. More importantly, pegylated TNT-b10 LLNs is stable for over four weeks and is over 10-fold more efficient than that of its counterpart TNT-a10 LLNs. Additionally, the optimal formulation O-TNT-b10 LLNs is capable of delivering mRNA encoding luciferase in vivo. These results provide useful insights into the design of next generation LLNs for mRNA delivery.

Advanced Search

close
  • Publications
  • Application Notes
  • Posters
  • Workshops
  • Videos & Webinars
  • Blog Posts
Search

Browse by Category

  • Application
    • Diagnostic and Imaging
    • Genetic Medicine
    • Hematology
    • Metabolic Disorders
    • Neuroscience
    • Oncology
    • Skeletal Disorders
    • Targeted Drug Delivery
    • Vaccines
    • Other Applications
  • Formulation
    • Liposomes
    • Nucleic Acid Lipid Nanoparticles
    • Polymeric Nanoparticles
    • Other Formulations
  • Payload
    • DNA
    • microRNA
    • mRNA
    • siRNA
    • Small Molecule Drugs
    • Other Payloads


related content

Publication - Summary

Effective Lipidoid Nanoparticle Delivery In Vivo of siRNA Targeting Kappa Light Chain Production in a Murine Xenograft Model

Xun Ma, Ping Zhou, Adin Kugelmass, Denis Toskic, Melissa Warner, Lisa X. Lee, Teresa Fogaren, Ming Wang, Yamin Li, Liu Yang, Qiaobing Xu and Raymond Comenzo

Researchers at Tufts University have recently reported a proof of concept for siRNA-LNP treatment for AL amyloidosis - a bone marrow disorder where Immunoglobulin (Ig) light chains (LC) misfold into amyloids that deposit in a variety of other tissues where they cause disfunction ...
Read More


Publication - Summary

MicroRNAs Enable mRNA Therapeutics to Selectively Program Cancer Cells to Self-Destruct

R. Jain, JP. Frederick, EY. Huang, KE. Burke, DM. Mauger, EA. Andrianova, SJ. Farlow, S. Siddiqui, J. Pimentel, K. Cheung-Ong, KM. McKinney, C. Köhrer, MJ. Moore, and T. Chakraborty

The advent of therapeutic mRNAs significantly increases the possibilities of protein-based biologics beyond those that can be synthesized by recombinant technologies (eg, monoclonal antibodies, extracellular enzymes, and cytokines). In addition to their application in the areas o...
Read More


Stay Informed

Sign up today to automatically receive new Precision NanoSystems application notes, conference posters, relevant science publications, and webinar invites.

MENU
菜单
X
X