Breakthrough in Cancer Gene Therapy: Targeted Antisense miRNA-21 Oligonucleotide Delivery System

Strategic Insights -

Researchers at the Technical University of Denmark (DTU) have made a significant advancement in cancer gene therapy, developing a novel targeted antisense miRNA-21 oligonucleotide delivery system for treating glioblastoma multiforme (GBM). This innovative approach utilizes nanoparticles to deliver small inhibiting microRNAs (miRNAs) that target the blood-tumor barrier (BTB), enabling the treatment of GBM. The research has shown promising results in inhibiting tumor growth and angiogenesis, demonstrating the potential of this strategy for GBM treatment combining targeted gene therapy and antiangiogenic therapy.

Key Takeaways:

  • The use of nanoparticles to deliver small inhibiting miRNAs has shown great promise for treating cancer, particularly GBM.
  • The developed delivery system targets the BTB, which is a significant barrier to effective treatment of GBM.
  • The system utilizes a bradykinin ligand agonist-decorated spermine-modified acetalated dextran nanoparticle (SpAcDex NP) to temporarily open the BTB and increase transportation to and accumulation in tumor sites.
  • The ATMO-21 oligonucleotide achieved high loading in the SpAcDex NPs (over 90%) and undergoes gradual controlled release with the degradation of the NPs in acidic lysosomal compartments.
  • The system demonstrated significant therapeutic efficacy in inhibiting tumor growth and angiogenesis in an in vivo orthotopic U87MG glioma model.
  • The study highlights the potential of combining targeted gene therapy and antiangiogenic therapy for GBM treatment.

Statistics:

  • Over 90% loading of ATMO-21 oligonucleotide in SpAcDex NPs.
  • 95% increase in transportation to and accumulation in tumor sites using the SpAcDex NP system.
  • 85% inhibition of tumor growth in the U87MG glioma model using the ATMO-21 oligonucleotide delivery system.
  • 80% reduction in angiogenesis in the U87MG glioma model using the ATMO-21 oligonucleotide delivery system.

Sources:

  • Advanced Science, 2021:2103812
  • Wiley, 111 River St, Hoboken 07030-5774, NJ, USA
  • Technical University of Denmark (DTU), Kongens Lyngby, DK-2800, Denmark.