Breakthrough in Cancer Gene Therapy: Nanoliposomes May Overcome Blood-Brain Barrier Obstacle

Strategic Insights -

Researchers from the Kermanshah University of Medical Sciences have made a significant discovery in cancer gene therapy, addressing a major challenge in delivering oligonucleotides to the brain, where neurodegenerative diseases occur. The team developed a nanoliposome-based delivery system, dubbed DNZ-NLP, which can effectively transport DNAzymes across the blood-brain barrier. This breakthrough has the potential to revolutionize the treatment of diseases such as Alzheimer's and Huntington's.

Key Takeaways:

  • The researchers generated a DNAzyme-loaded nanoliposome (DNZ-NLP) to deliver oligonucleotides to the brain, overcoming the primary obstacle of the blood-brain barrier.
  • The DNZ-NLPs had an optimum size of ~ 68 nm for brain delivery and demonstrated stable biphasic release profiles in 48 hours.
  • Cellular uptake and cytocompatibility studies showed that both blank nanoliposomes (B-NLPs) and DNZ-NLPs were cytocompatible.
  • About 60% of intravenously administered DNZ-NLPs to healthy mice were found in the brains of the animals, successfully crossing the BBB.
  • The controlled release of DNAzymes and improved BBB permeability suggest that DNZ-NLPs offer a promising formulation for delivering oligonucleotides to the brain.

Statistics:

  • 68 nm: The optimum size of DNZ-NLPs for brain delivery.
  • 48 hours: The time frame of the biphasic release profile of DNZ-NLPs.
  • 60%: The percentage of DNZ-NLPs found in the brains of healthy mice after intravenous administration.
  • 15(1): The issue and volume of the Scientific Reports journal in which the research was published.

Sources:

  • Facilitating DNAzyme transport across the blood-brain barrier with nanoliposome technology. Scientific Reports, 2025;15(1):18914.
  • Nature Portfolio, Heidelberger Platz 3, Berlin, 14197, Germany (Publisher's contact information for the journal Scientific Reports).
  • Mohammad Javad Hoseinifar, Nano Drug Delivery Research Center, Faculty of Pharmacy, Kermanshah University of Medical Sciences, Kermanshah, Iran (Correspondent's contact information).
  • Kermanshah University of Medical Sciences Reports Findings in Cancer Gene Therapy (Facilitating DNAzyme transport across the blood-brain barrier with nanoliposome technology). Journal of Engineering. June 9, 2025; p 1519 (Citation for the news report).