Understanding Frontotemporal Dementia and ALS to Find New Treatments
Frontotemporal dementia and related disorders: transcriptomic profiling, biomarker discovery, and mechanistic insight
This project aims to find new ways to identify and treat frontotemporal dementia and amyotrophic lateral sclerosis by looking closely at genetic information in affected brain tissue.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Mayo Clinic Jacksonville NIH-funded |
| Lab location | 1 site (Jacksonville, United States) |
| Project ID | NIH-11080976 on NIH RePORTER |
What this research studies
We are exploring the genetic changes in brain tissue from patients with frontotemporal dementia (FTD) and amyotrophic lateral sclerosis (ALS), two related and severe brain conditions. Our team uses advanced long-read sequencing technology to get a very detailed look at how genes are working, especially focusing on how RNA is processed. By examining specific brain cells, we hope to uncover unique genetic signatures that contribute to these diseases. This deep dive into the genetic makeup of affected cells could reveal new ways to diagnose and develop treatments for FTD and ALS.
Who could benefit from this research
Good fit: Patients with frontotemporal dementia or amyotrophic lateral sclerosis, or those with a family history of these conditions, may benefit from the future treatments developed from this foundational research.
Not a fit: Patients without frontotemporal dementia or amyotrophic lateral sclerosis would not directly benefit from this specific research.
Why it matters
Potential benefit: If successful, this work could lead to new ways to diagnose frontotemporal dementia and ALS earlier and help develop new medications to slow or stop disease progression.
How similar studies have performed: While the role of RNA processing in neurodegenerative diseases is an active area of investigation, this specific approach using full-length long-read transcriptomic data in FTD-ALS patient tissue is a novel and advanced method.
Where this research is happening
Jacksonville, United States
- Mayo Clinic Jacksonville — Jacksonville, United States (Active)
Researchers
- Principal investigator: Van Blitterswijk, Marka — Mayo Clinic Jacksonville
- Study coordinator: Van Blitterswijk, Marka
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.