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Protection against lethal Marburg virus infection mediated by lipid encapsulated small interfering RNA

Raul Ursic-Bedoya1, Chad E Mire, Marjorie Robbins

  • 1Tekmira Pharmaceuticals, Burnaby, British Columbia, Canada.

The Journal of Infectious Diseases
|August 31, 2013
PubMed
Abstract

Insights

Lipid-encapsulated small interfering RNA (siRNA) shows promise in treating Marburg virus (MARV) infection. Studies demonstrated significant survival rates in animal models, highlighting siRNA as a potential therapeutic for MARV.

Area of Science:

  • Virology
  • RNA Therapeutics
  • Nanotechnology

Background:

  • Marburg virus (MARV) infection poses a significant threat, causing severe illness and death in humans and primates.
  • No approved therapeutics currently exist for MARV infection.
  • This study explores small interfering RNA (siRNA) encapsulated in lipid nanoparticles as a potential treatment.

Purpose of the Study:

  • To develop and evaluate lipid-encapsulated siRNA for Marburg virus treatment.
  • To assess the in vitro and in vivo efficacy of anti-MARV siRNAs.

Main Methods:

  • siRNA activity was confirmed using dual luciferase reporter assays and live virus testing.
  • Lead siRNA candidates were tested in guinea pig models infected with three MARV strains (Angola, Ci67, Ravn).

Main Results:

  • siRNA treatment achieved 60%-100% survival rates in guinea pigs.
  • Targeting MARV nucleoprotein (NP) mRNA, specifically NP-718m siRNA in lipid nanoparticles, yielded the highest survival rates.
  • NP-718m provided 100% protection against MARV Angola and Ci67 strains, and 60% against Ravn. A combination of NP-718m and NP-143m achieved 100% protection against MARV Ravn.

Conclusions:

  • The developed lipid nanoparticle-encapsulated siRNA demonstrated protective efficacy against highly pathogenic MARV strains.
  • Further development of this lipid nanoparticle technology holds potential for effective MARV infection treatments.
Keywords:
LNPMarburg virusRNAifilovirusguinea pigsiRNAtherapeuticstreatment

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