Investigating the role of CGG repeats in Fragile X disorders and potential therapies
The FMR1 CGG repeat as functional element and therapeutic target in Fragile X associated disorders
This study is looking at how certain genetic repeats in the FMR1 gene affect brain function in people with Fragile X Syndrome, with the goal of finding new ways to help improve their symptoms and treatment options.
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-10907683 on NIH RePORTER |
What this research studies
This research explores the functional role of CGG repeats in the FMR1 gene, which is linked to Fragile X Syndrome and related disorders. It aims to understand how these repeats affect protein synthesis and contribute to neurological conditions. By utilizing advanced techniques like CRISPR and antisense oligonucleotides, the study seeks to develop therapeutic strategies that could alleviate the symptoms associated with these disorders. Patients may benefit from insights into new treatment options that target the underlying genetic mechanisms.
Who could benefit from this research
Good fit: Ideal candidates for participation are individuals diagnosed with Fragile X Syndrome or Fragile X-associated Tremor/Ataxia Syndrome.
Not a fit: Patients without a diagnosis of Fragile X-related disorders or those with unrelated neurological conditions may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to innovative therapies that improve outcomes for individuals with Fragile X Syndrome and related conditions.
How similar studies have performed: Previous research has shown promise in targeting genetic mechanisms in neurological disorders, suggesting potential for success in this novel approach.
Where this research is happening
Houston, United States
- Baylor College of Medicine — Houston, United States (Active)
Researchers
- Principal investigator: Todd, Peter K — Baylor College of Medicine
- Study coordinator: Todd, Peter K
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.