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Characterization of Functionally Associated miRNAs in Glioblastoma and their Engineering into Artificial Clusters for Gene Therapy
Published on: October 4, 2019
Current progress for the use of miRNAs in glioblastoma treatment
Amanda Tivnan1, Kerrie Leanne McDonald
1Cure for Life Neuro-Oncology Group, Lowy Cancer Research Centre, Prince of Wales Clinical School, University of New South Wales, Sydney, 2052, Australia, a.tivnan@unsw.edu.au.
Abstract:
Glioblastoma (GBM) is a highly aggressive brain cancer with the worst prognosis of any central nervous system disease despite intensive multimodal therapy. Inevitably, glioblastoma is fatal, with recurrence of treatment-resistant tumour growth at distal sites leading to an extremely low median survival rate of 12-15 months from the time of initial diagnosis. With the advent of microarray and gene profiling technology, researchers have investigated trends in genetic alterations and, in this regard, the role of dysregulated microRNAs (highly conserved endogenous small RNA molecules) in glioblastoma has been studied with a view to identifying novel mechanisms of acquired drug resistance and allow for development of microRNA (miRNA)-based therapeutics for GBM patients. Considering the development of miRNA research from initial association to GBM to commercial development of miR-based therapeutics in less than a decade, it is not beyond reasonable doubt to anticipate significant advancements in this field of study, hopefully with the ultimate conclusion of improved patient outcome. This review discusses the recent advancements in miRNA-based therapeutic development for use in glioblastoma treatment and the challenges faced with respect to in vivo and clinical application.
Insights
MicroRNAs (miRNAs) are small RNA molecules being investigated for glioblastoma (GBM) treatment. Research aims to develop novel miRNA-based therapies to overcome drug resistance and improve patient outcomes for this aggressive brain cancer.
Area of Science:
- Oncology
- Molecular Biology
- Genetics
Background:
- Glioblastoma (GBM) is a fatal brain cancer with poor prognosis despite treatment.
- Recurrent, treatment-resistant GBM limits patient survival to 12-15 months.
- MicroRNAs (miRNAs) are small RNA molecules implicated in GBM development and drug resistance.
Purpose of the Study:
- To review recent advancements in miRNA-based therapeutic development for GBM.
- To identify novel mechanisms of acquired drug resistance in GBM mediated by miRNAs.
- To discuss challenges in the clinical application of miRNA therapeutics for glioblastoma.
Main Methods:
- Review of current scientific literature on miRNA research in glioblastoma.
- Analysis of gene profiling and microarray data related to miRNA dysregulation in GBM.
- Evaluation of preclinical and clinical studies on miRNA-based therapeutics for GBM.
Main Results:
- miRNA research has rapidly progressed from initial association to therapeutic development for GBM.
- Dysregulated miRNAs play a significant role in GBM pathogenesis and drug resistance.
- Several miRNA-based therapeutic strategies are under investigation for glioblastoma treatment.
Conclusions:
- Targeting miRNAs offers a promising avenue for novel glioblastoma therapies.
- Overcoming challenges in in vivo and clinical application is crucial for miRNA therapeutics.
- Advancements in miRNA research hold potential for improving glioblastoma patient outcomes.
