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Related Experiment Videos

Antisense oligonucleotides as therapeutic agents.

U Galderisi1, A Cascino, A Giordano

  • 1Department of Pathology, Jefferson Medical College of Thomas Jefferson University, Philadelphia, Pennsylvania, USA.

Journal of Cellular Physiology
|September 25, 1999
PubMed
Summary

Antisense oligonucleotides offer a promising therapeutic strategy by blocking target gene expression for various diseases. Clinical trials show safety and efficacy in treating cancers, cardiovascular disorders, and viral infections like HIV and CMV.

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Area of Science:

  • Molecular Biology
  • Genetics
  • Pharmacology

Background:

  • Antisense oligonucleotides (ASOs) are synthetic nucleic acid molecules designed to selectively inhibit gene expression.
  • Their therapeutic potential is being explored across diverse medical fields, including oncology, cardiology, and virology.
  • Challenges in efficacy and reliability have historically tempered enthusiasm for ASO therapeutics.

Purpose of the Study:

  • To review the current status and therapeutic applications of antisense oligonucleotides.
  • To highlight preliminary clinical findings demonstrating the safety and efficacy of ASOs in treating various human diseases.
  • To assess the potential of ASO-based therapies in oncology, cardiovascular disorders, and viral infections.

Main Methods:

  • Review of preclinical and clinical studies on antisense oligonucleotide therapies.

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  • Analysis of data from ongoing clinical trials for cancer, cardiovascular diseases, and viral infections.
  • Examination of the safety and pharmacokinetic profiles of phosphorothioate oligonucleotides.

    • Main Results:

    • Preliminary clinical studies indicate that antisense oligodeoxynucleotides (ODNs) are safe and effective in treating malignant diseases, including ovarian cancer and hematological malignancies.
    • ASO therapy has shown utility in managing cardiovascular conditions like restenosis and graft occlusion.
    • Antisense oligonucleotides are emerging as promising antiviral agents, with ongoing trials targeting HIV-1 and hepatitis viruses.

    Conclusions:

    • Antisense oligonucleotide technology demonstrates significant therapeutic promise across multiple disease areas.
    • Phosphorothioate ODNs have shown acceptable safety and pharmacokinetic profiles in Phase I and II trials, supporting further development.
    • The development of drugs like Vitravene for cytomegalovirus (CMV) infections signifies substantial progress in antisense-based therapeutic strategies.