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Targeting miRNAs and Other Non-Coding RNAs as a Therapeutic Approach: An Update
Emine Bayraktar1,2, Recep Bayraktar2,3, Hulya Oztatlici4,5
1Department of Gynecologic Oncology, The University of Texas MD Anderson Cancer Center, Houston, TX 77030, USA.
Abstract:
Since the discovery of the first microRNAs (miRNAs, miRs), the understanding of miRNA biology has expanded substantially. miRNAs are involved and described as master regulators of the major hallmarks of cancer, including cell differentiation, proliferation, survival, the cell cycle, invasion, and metastasis. Experimental data indicate that cancer phenotypes can be modified by targeting miRNA expression, and because miRNAs act as tumor suppressors or oncogenes (oncomiRs), they have emerged as attractive tools and, more importantly, as a new class of targets for drug development in cancer therapeutics. With the use of miRNA mimics or molecules targeting miRNAs (i.e., small-molecule inhibitors such as anti-miRS), these therapeutics have shown promise in preclinical settings. Some miRNA-targeted therapeutics have been extended to clinical development, such as the mimic of miRNA-34 for treating cancer. Here, we discuss insights into the role of miRNAs and other non-coding RNAs in tumorigenesis and resistance and summarize some recent successful systemic delivery approaches and recent developments in miRNAs as targets for anticancer drug development. Furthermore, we provide a comprehensive overview of mimics and inhibitors that are in clinical trials and finally a list of clinical trials based on miRNAs.
Insights
MicroRNAs (miRNAs) are key regulators in cancer development and progression. Targeting miRNAs with mimics or inhibitors shows promise for novel anticancer drug development and therapeutics.
Area of Science:
- Oncology
- Molecular Biology
- Genetics
Background:
- MicroRNAs (miRNAs) are small non-coding RNAs that regulate gene expression.
- miRNAs play critical roles in fundamental biological processes, including cancer hallmarks like proliferation, survival, and metastasis.
- Dysregulated miRNA expression is implicated in tumorigenesis and cancer resistance.
Purpose of the Study:
- To discuss the role of miRNAs and other non-coding RNAs in cancer.
- To summarize recent advances in miRNA-targeted anticancer drug development.
- To provide an overview of miRNA-based therapeutics in clinical trials.
Main Methods:
- Review of experimental data and literature on miRNA biology in cancer.
- Analysis of miRNA mimics and inhibitors as therapeutic strategies.
- Compilation of clinical trial data for miRNA-targeted therapies.
Main Results:
- miRNAs function as tumor suppressors or oncogenes (oncomiRs), influencing cancer phenotypes.
- miRNA-based therapeutics, including mimics (e.g., miRNA-34) and inhibitors (anti-miRs), show preclinical promise.
- Several miRNA-targeted drugs have advanced to clinical development for cancer treatment.
Conclusions:
- miRNAs are crucial regulators in cancer and represent a new class of therapeutic targets.
- Targeting miRNA expression offers a promising strategy for developing novel anticancer drugs.
- Ongoing clinical trials highlight the therapeutic potential of miRNA-based approaches in oncology.
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