Targeting disorders related to the MeCP2 protein using specific oligonucleotides
Official title: Targeting MeCP2-Associated Disorders with miRNA Site-Blocking Oligonucleotides
Funded at Loyola University Chicago · NIH-11115552 · Data updated 2026-11-12
This study is looking at a new way to help people with Rett syndrome by using special tools to adjust a protein in the brain that’s important for their development, with the hope of improving their symptoms.
Quick facts
| Grant type | Fellowship grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Loyola University Chicago NIH-funded |
| Lab location | 1 site (Maywood, United States) |
| Project start | May 1, 2024 |
| Project end | April 30, 2027 |
| Funding year | 2025 |
| Award (this year) | $80,440 |
| NIH project number | 5F32HD113386-02 |
| Project ID | NIH-11115552 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Rett syndrome or related MeCP2-associated disorders.
✕Less likely to help
Patients with other neurodevelopmental disorders not associated with MeCP2 mutations may not benefit from this research.
What this research studies
This research focuses on Rett syndrome, a neurodevelopmental disorder caused by mutations in the MeCP2 protein. The approach involves using miRNA site-blocking oligonucleotides to regulate the expression of MeCP2, aiming to restore its levels in the brain. By fine-tuning the gene expression through the 3' untranslated region of mRNA, the research seeks to develop a therapeutic strategy that could potentially reverse the effects of the disorder. The methodology includes careful delivery of these oligonucleotides to ensure consistent dosing across brain cells.
Why it matters
Potential benefit: If successful, this research could lead to new therapeutic options for patients with Rett syndrome, potentially reversing some of the disorder's effects.
How similar studies have gone: Previous studies have shown promise in using similar approaches to regulate gene expression in animal models, suggesting potential for success in human applications.
Where this research is happening
-
Loyola University Chicago
Maywood, United States Active
Researchers
- Principal investigator: Vanderplow, Amanda — Loyola University Chicago
- Study coordinator: Vanderplow, Amanda
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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.