MicroRNA pathways in Angelman Syndrome: novel targets for therapy
Official title: MicroRNA mechanism of Angelman Syndrome
Funded at Ohio State University · NIH-11453242 · Data updated 2026-11-12
A small study explores how specific microRNAs may influence Angelman syndrome and whether targeting them could lead to new treatments.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Ohio State University NIH-funded |
| Lab location | 1 site (Columbus, UNITED STATES) |
| Project start | September 1, 2026 |
| Project end | August 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $426,914 |
| NIH project number | 1R21HD119646-01A1 |
| Project ID | NIH-11453242 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates would be individuals with Angelman syndrome or related neurodevelopmental features who may benefit from future targeted therapies.
✕Less likely to help
People without Angelman syndrome or those not involved in future translational trials may not benefit.
What this research studies
Angelman syndrome is a severe neurodevelopmental condition with no cure, and this grant looks at how tiny RNA molecules called microRNAs might drive the disease. The researchers examined brain tissue from animal models to identify a miRNA cluster (miR-183/96/182) that is reduced in AS. They are exploring whether manipulating these microRNAs could correct neural signaling and improve symptoms, building on previous work where cannabidiol showed some benefits in AS models. The project uses focused molecular analyses and experiments in animal systems to understand whether these microRNAs could become treatment targets for AS in humans.
Why it matters
Potential benefit: If successful, this work could point to new molecular targets for therapies that might reduce epilepsy, improve brain function, or lessen symptoms in Angelman syndrome.
How similar studies have gone: Other studies in AS and neurological disorders have shown that microRNA pathways can influence brain function and disease symptoms, but this exact miR-183/96/182 cluster in AS is still under investigation.
Where this research is happening
-
Ohio State University
Columbus, United States Active
Researchers
- Principal investigator: Gu, Bin — Ohio State University
- Study coordinator: Gu, Bin
Related research
Other NIH-funded projects on the same topics.
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Genome editor therapy for Angelman syndrome in young children
A University of North Carolina team is exploring a gene-editing approach to turn on the silent UBE3A gene in people with Angelman syndrome to reduce symptoms.
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Delta brain rhythms in Angelman syndrome
Scientists will study unusual brain wave patterns in Angelman syndrome to understand how these signals relate to behavior and guide future treatments for affected children and adults.
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Targeting the stress response in Angelman syndrome
Aims to understand how loss of a brain gene called UBE3A disrupts pathways in Angelman syndrome and to design therapies that calm the stress response in affected neurons.
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Small molecule therapy to turn on the paternal UBE3A gene in Angelman syndrome
A small molecule drug is being developed to turn on the paternal UBE3A gene in Angelman syndrome, with the goal of improving brain function.
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UBE3A mosaicism and Angelman syndrome phenotypes
A study from UNC Chapel Hill will explore how the mix of neurons with active paternal UBE3A affects Angelman syndrome symptoms using a special mouse model and gene therapy tools.
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How body‑sensing nerves affect walking in Angelman syndrome
This project looks at whether problems with a nerve sensor called PIEZO2 cause balance and walking problems in people with Angelman syndrome.
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.