Developing a new treatment for Angelman Syndrome using modified RNA technology
Official title: Engineering Chemically Modified RNPs for Effective Treatment of Angelman Syndrome
Funded at Couragene, INC. · NIH-10918729 · Data updated 2026-11-12
This study is testing a new way to treat Angelman Syndrome by using special tools to help turn on a gene that’s not working properly in the brain, with the hope of improving development for those affected.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Couragene, INC. NIH-funded |
| Lab location | 1 site (Edison, United States) |
| Project start | September 20, 2024 |
| Project end | August 31, 2025 |
| Funding year | 2024 |
| Award (this year) | $339,999 |
| NIH project number | 1R41NS134480-01A1 |
| Project ID | NIH-10918729 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Angelman Syndrome, particularly those with the genetic deficiency of the UBE3A gene.
✕Less likely to help
Patients with other neurogenetic disorders or those without a confirmed diagnosis of Angelman Syndrome may not benefit from this research.
What this research studies
This research focuses on creating a novel treatment for Angelman Syndrome, a genetic disorder that leads to severe developmental delays. The approach involves using chemically modified ribonucleoproteins (cRNPs) to deliver CRISPR technology directly to brain cells, aiming to activate the UBE3A gene that is deficient in patients. By optimizing the formulation of these cRNPs, the researchers hope to achieve high efficiency in editing the genes of affected neuronal cells. This innovative method seeks to provide a safer alternative to current gene editing techniques that may have safety concerns.
Why it matters
Potential benefit: If successful, this research could lead to a groundbreaking treatment that addresses the underlying cause of Angelman Syndrome rather than just managing its symptoms.
How similar studies have gone: Previous research has shown promise in using CRISPR technology for gene editing in similar contexts, indicating potential success for this novel approach.
Where this research is happening
-
Couragene, INC.
Edison, United States Active
Researchers
- Principal investigator: Xie, Ying — Couragene, INC.
- Study coordinator: Xie, Ying
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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MicroRNA pathways in Angelman Syndrome: novel targets for therapy
A small study explores how specific microRNAs may influence Angelman syndrome and whether targeting them could lead to new treatments.
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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.
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.