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MicroRNAs in cancer: from developmental genes in worms to their clinical application in patients
11] Department of Experimental Therapeutics, The University of Texas, MD Anderson Cancer Center, Houston, TX, USA [2] Division of Oncology, Medical University of Graz, Austria.
Abstract:
Several discoveries have paved the way to personalise cancer medicine and a tremendous gain of knowledge in genomics and molecular mechanisms of cancer progression cumulated over the last years. Big stories in biology commonly start in a simple model system. No wonder microRNAs have been identified as regulators of embryonic development in the nematode Caenorhabditis elegans. From the first identification in worms to the first-in-man microRNA-based clinical trial in humans, almost 20 years passed. In this review we follow the story of understanding microRNA alterations in cancer, describe recent developments in the microRNA field and critically discuss their potential as diagnostic, prognostic and therapeutics factors in cancer medicine. We will explain the rationale behind the use of microRNAs in cancer diagnosis and prognosis prediction, but also discuss the limitations and pitfalls associated with this. Novel developments of combined microRNA/siRNA pharmacological approaches will be discussed and most recently data about MXR34, the first-tested microRNA drug will be described.
Insights
MicroRNAs, small RNA molecules regulating gene expression, are key to personalized cancer medicine. This review explores their role in cancer diagnosis, prognosis, and therapy, including novel drug developments.
Area of Science:
- Molecular Biology
- Genomics
- Cancer Research
Background:
- Recent advances in genomics and molecular biology have enabled personalized cancer medicine.
- MicroRNAs were first identified as regulators of embryonic development in Caenorhabditis elegans.
- The field has progressed from basic discovery to clinical applications in under two decades.
Purpose of the Study:
- To review the understanding of microRNA alterations in cancer.
- To discuss recent developments in microRNA research.
- To critically evaluate microRNAs as diagnostic, prognostic, and therapeutic factors in oncology.
Main Methods:
- Literature review of microRNA research in cancer.
- Analysis of recent developments in microRNA diagnostics and therapeutics.
- Discussion of clinical trial data and novel drug approaches.
Main Results:
- MicroRNAs play significant roles in cancer progression and can be altered in various cancers.
- MicroRNAs show potential as biomarkers for cancer diagnosis and prognosis.
- Novel pharmacological strategies involving microRNAs are emerging, with early clinical data available.
Conclusions:
- MicroRNAs hold considerable promise for personalized cancer medicine, serving as diagnostic, prognostic, and therapeutic targets.
- Understanding the limitations and potential pitfalls is crucial for clinical translation.
- Emerging microRNA-based drugs, like MXR34, represent a new frontier in cancer treatment.

