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Antisense DNAs as targeted genetic medicine to treat cancer
1Cellular Biochemistry Section, Basic Research Laboratory, National Cancer Institute, NIH, Bethesda, MD 20892-1750, USA. chochung@helix.nih.gov
Abstract:
Nucleic acid therapies represent a direct genetic approach for cancer treatment. Such an approach takes advantage of mechanisms that activate genes known to confer a growth advantage to neoplastic cells. The ability to block the expression of these genes allows exploration of normal growth regulation. Progress in antisense technology has been rapid, and the traditional antisense inhibition of gene expression is now viewed on a genomic scale. This global view has led to a new vision in antisense technology, the elimination of nonspecific and undesirable side effects, and ultimately, the generation of more effective and less toxic nucleic acid medicines. Several antisense oligonucleotides are in clinical trials, are well tolerated, and are potentially active therapeutically. Antisense oligonucleotides are promising molecular medicines for treating human cancer in the near future.
Insights
Nucleic acid therapies offer a direct genetic approach to cancer treatment by blocking cancer-promoting genes. Advances in antisense technology are leading to more effective and less toxic nucleic acid medicines for future cancer care.
Area of Science:
- Oncology
- Genetics
- Molecular Biology
Background:
- Nucleic acid therapies provide a direct genetic strategy for cancer treatment.
- Antisense technology has evolved from traditional gene expression inhibition to a genomic scale approach.
- This evolution aims to reduce side effects and enhance the efficacy of nucleic acid medicines.
Purpose of the Study:
- To explore the potential of nucleic acid therapies in cancer treatment.
- To highlight advancements in antisense technology for improved cancer therapeutics.
- To discuss the development of more effective and less toxic nucleic acid medicines.
Main Methods:
- Focus on blocking gene expression that confers a growth advantage to neoplastic cells.
- Leveraging advancements in antisense technology for genomic-scale gene inhibition.
- Clinical evaluation of antisense oligonucleotides for therapeutic activity and tolerability.
Main Results:
- Antisense technology is now viewed on a genomic scale, enabling a broader application.
- This global perspective facilitates the elimination of nonspecific and undesirable side effects.
- Several antisense oligonucleotides are in clinical trials, demonstrating good tolerability and potential therapeutic activity.
Conclusions:
- Nucleic acid therapies represent a promising frontier in cancer treatment.
- Antisense oligonucleotides are advancing towards becoming effective and less toxic molecular medicines.
- These therapies hold significant promise for treating human cancers in the near future.