m6A RNA modifications and FXTAS: uncovering new paths to treatment

Who could benefit from this research

What this research studies

FXTAS is a late-onset neurodegenerative condition affecting people who carry a CGG premutation in the FMR1 gene. This grant investigates how m6A RNA modifications in the brain might drive or worsen neurodegeneration in FXTAS, using focused laboratory studies and brain-related tissue analyses. By mapping m6A-related changes and identifying affected molecular pathways, the project aims to reveal new targets for therapies that could help patients. The work builds on preliminary findings suggesting m6A regulation is altered in FXTAS and seeks to translate those insights toward clinically relevant strategies.

Why it matters

Where this research is happening

• Emory University
  Atlanta, United States Active

Researchers

• Principal investigator: Shafik, Andrew Mark — Emory University
• Study coordinator: Shafik, Andrew Mark

Related research

Other NIH-funded projects on the same topics.

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  Researchers will follow people who carry FMR1 premutations—some with FXTAS, some without, and healthy volunteers—over two years to find brain, movement, and molecular markers linked to disease changes.

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About this research

1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
3. For full project details, budget, and progress reports, visit the official NIH RePORTER page.