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Highly Efficient Ligation of Small RNA Molecules for MicroRNA Quantitation by High-Throughput Sequencing
Published on: November 18, 2014
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Small Molecule Regulators of microRNAs Identified by High-Throughput Screen Coupled with High-Throughput Sequencing
Anna Krichevsky1, Lien Nguyen1, Zhiyun Wei1
1Brigham and Women's Hospital and Harvard Medical School.
Research Square
|March 30, 2023
Summary
Small molecules can modulate microRNAs (miRNAs) to treat diseases. Researchers found cardiac glycosides induce miR-132, protecting neurons in Alzheimer's disease models.
Area of Science:
- Biochemistry
- Neuroscience
- Genetics
Background:
- MicroRNAs (miRNAs) are crucial regulators of gene expression, controlling fundamental biological processes.
- Dysregulation of miRNAs is implicated in numerous diseases, presenting therapeutic opportunities.
- Current miRNA modulation strategies face challenges, particularly for neurological disorders, lacking clinical approval.
Approach:
- A biodiverse library of small molecule compounds was screened for miRNA modulation.
- Screening was performed in human induced pluripotent stem cell-derived neurons.
- The study identified cardiac glycosides as potent modulators of specific miRNAs.
Key Points:
- Cardiac glycosides were identified as potent inducers of miR-132.
- miR-132 is a key miRNA downregulated in Alzheimer's disease and tauopathies.
- Cardiac glycosides downregulated miR-132 targets, including Tau, and offered neuroprotection.
Conclusions:
- Small molecules offer a promising alternative for miRNA-based therapeutics.
- Cardiac glycosides demonstrate potential for treating neurological diseases like Alzheimer's.
- The generated dataset of compound effects on the miRNome serves as a resource for future drug discovery.
