Investigating biomarkers for MeCP2-related diseases in children
Comprehensive Deep Phenotyping and Multi-omics to Develop Clinical and Molecular Biomarkers for MeCP2-related Diseases
This study is looking at the genes and molecules involved in Rett Syndrome and MECP2 duplication syndrome to find better ways to treat children with these conditions, making sure the treatments work well and are safe for them.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Baylor College of Medicine NIH-funded |
| Lab location | 1 site (Houston, United States) |
| Project ID | NIH-10877193 on NIH RePORTER |
What this research studies
This research focuses on understanding the genetic and molecular factors associated with MeCP2-related diseases, specifically Rett Syndrome and MECP2 duplication syndrome, which affect children. By utilizing comprehensive deep phenotyping and multi-omics approaches, the study aims to develop clinical and molecular biomarkers that can help tailor treatments for these conditions. The research will involve analyzing biological samples and assessing treatment responses to ensure that therapies are effective without causing adverse effects. This approach is crucial for managing the delicate balance of MeCP2 levels in patients.
Who could benefit from this research
Good fit: Ideal candidates for this research are children aged 0-11 years diagnosed with Rett Syndrome or MECP2 duplication syndrome.
Not a fit: Patients who do not have MeCP2-related diseases or are outside the age range of 0-11 years may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to more effective and personalized treatments for children with MeCP2-related diseases.
How similar studies have performed: Previous research has shown promising results with antisense oligonucleotide therapies in animal models, indicating potential for success in human applications.
Where this research is happening
Houston, United States
- Baylor College of Medicine — Houston, United States (Active)
Researchers
- Principal investigator: Pehlivan, Davut — Baylor College of Medicine
- Study coordinator: Pehlivan, Davut
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 below.