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Author Spotlight: Innovative Cancer Therapies with Iron Oxide Nanoparticles for Glioblastoma Treatment
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Clinical applications of oligonucleotides for cancer therapy

Vittorio DeFranciscis1, Giovanni Amabile2, Marcin Kortylewski3

  • 1National Research Council, Institute of Genetic and Biomedical Research, Milan, Italy.

Molecular Therapy : the Journal of the American Society of Gene Therapy
|March 6, 2025
PubMed

Insights

Oligonucleotide therapeutics (ONTs) are revolutionizing cancer treatment by precisely modulating gene expression. Despite delivery challenges, ONTs offer new hope for targeting difficult molecules and enhancing immunotherapy.

Area of Science:

  • Biotechnology
  • Molecular Biology
  • Oncology

Background:

  • Oligonucleotide therapeutics (ONTs) are a rapidly advancing class of drugs for cancer treatment.
  • Over 20 nucleic acid-based therapies are approved, showcasing significant progress in chemical modifications, sequence optimization, and delivery systems.
  • ONTs offer novel strategies for targeting previously undruggable molecules and enhancing cancer immunotherapy.

Purpose of the Study:

  • To provide a comprehensive review of oligonucleotide therapeutics in clinical trials for cancer.
  • To highlight emerging delivery strategies and innovative therapeutic approaches.
  • To emphasize the role of ONTs in cancer immunotherapy and address challenges to clinical translation.

Main Methods:

  • Review of current literature on oligonucleotide therapeutics in oncology.
  • Analysis of clinical trial data and regulatory approvals.
  • Examination of advancements in delivery systems and therapeutic strategies.

Main Results:

  • Oligonucleotide therapeutics have shown promise in targeting specific genes and enhancing cancer immunotherapy.
  • Recent FDA approval for a myeloproliferative disease highlights the clinical potential of ONTs.
  • Key challenges include tissue-specific delivery, off-target effects, cellular uptake, and endosomal release.

Conclusions:

  • Oligonucleotide therapeutics hold significant potential to revolutionize oncology and improve patient outcomes.
  • Addressing current challenges in delivery and efficacy is crucial for broader clinical translation.
  • ONTs represent a powerful tool for personalized cancer medicine and overcoming treatment resistance.
Keywords:
antisense oligonucleotidesaptamerscancer therapydeliveryimmunotherapyoligonucleotidesiRNA

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