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Updated: May 21, 2026

Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells
Published on: August 25, 2021
New approaches for cancer treatment: antitumor drugs based on gene-targeted nucleic acids
O A Patutina1, N L Mironova, V V Vlassov
1Institute of Chemical Biology and Fundamental Medicine, Siberian Branch, Russian Academy of Sciences.
Abstract:
Currently, the main way to fight cancer is still chemotherapy. This method of treatment is at the height of its capacity, so, setting aside the need for further improvements in traditional treatments for neoplasia, it is vital to develop now approaches toward treating malignant tumors. This paper reviews innovational experimental approaches to treating malignant malformations based on the use of gene-targeted drugs, such as antisense oligonucleotides (asON), small interfering RNA (siRNA), ribozymes, and DNAzymes, which can all inhibit oncogene expression. The target genes for these drugs are thoroughly characterized, and the main results from pre-clinical and first-step clinical trials of these drugs are presented. It is shown that the gene-targeted oligonucleotides show considerable variations in their effect on tumor tissue, depending on the target gene in question. The effects range from slowing and stopping the proliferation of tumor cells to suppressing their invasive capabilities. Despite their similarity, not all the antisense drugs targeting the same region of the mRNA of the target-gene were equally effective. The result is determined by the combination of the drug type used and the region of the target-gene mRNA that it complements.
Insights
Gene-targeted drugs like antisense oligonucleotides show promise in treating cancer by inhibiting oncogene expression. Their effectiveness varies based on the specific target gene and mRNA region, impacting tumor proliferation and invasion.
Area of Science:
- Oncology
- Molecular Biology
- Biotechnology
Background:
- Chemotherapy remains the primary cancer treatment but has limitations.
- Novel therapeutic strategies are crucial for treating malignant tumors.
- Gene-targeted drugs offer innovative approaches to inhibit oncogene expression.
Purpose of the Study:
- To review experimental gene-targeted drugs for cancer treatment.
- To present pre-clinical and clinical trial results of these novel therapies.
- To analyze the efficacy of different gene-targeted drugs based on target genes.
Main Methods:
- Review of experimental approaches using gene-targeted drugs.
- Focus on antisense oligonucleotides (asON), small interfering RNA (siRNA), ribozymes, and DNAzymes.
- Analysis of pre-clinical and early-stage clinical trial data.
Main Results:
- Gene-targeted oligonucleotides demonstrated varied effects on tumor tissues.
- Observed effects included inhibition of tumor cell proliferation and suppression of invasiveness.
- Efficacy depended on the drug type and the specific mRNA target region.
Conclusions:
- Gene-targeted drugs represent a promising avenue for cancer therapy.
- The effectiveness of these drugs is highly specific to the target gene and mRNA sequence.
- Further research is needed to optimize these novel therapeutic strategies.
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