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


Authors: 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

Journal: Nucleic Acid Therapeutics

DOI: 10.1089/nat.2018.0734

Publication - Abstract

September 24, 2018

Abstract

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 of vaccine development, immune-oncology, and protein replacement therapies, one exciting possibility is to use therapeutic mRNAs to program undesired, diseased cells to synthesize a toxic intracellular protein, causing cells to self-destruct. For this approach to work, however, methods are needed to limit toxic protein expression to the intended cell type. Here, we show that inclusion of microRNA target sites in therapeutic mRNAs encoding apoptotic proteins, Caspase or PUMA, can prevent their expression in healthy hepatocytes while triggering apoptosis in hepatocellular carcinoma cells.

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

Lipid Nanoparticle Delivery of siRNA to Silence Neuronal Gene Expression in the Brain

Rungta RL, Choi HB, Lin PJ, Ko RW, Ashby D, Nair J, Manoharan M, Cullis PR, MacVicar BA

The ability to manipulate gene expression and regulation in neurons is an invaluable asset to advancement of neuroscience and developing therapeutics for neurological disorders. 

Read More


Publication - Abstract

Liposomes used for the delivery of pharmaceuticals have difficulties scaling up and reaching clinical translation as they suffer from batch-to-batch variability. 
Read More


Stay Informed

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

MENU
X