Identifying new treatment targets for ALS and frontotemporal dementia
Single-Cell Transcriptional and Epigenomic Dissection to Identify Therapeutic Targets for ALS and FTD
This study is looking at brain samples from people with ALS and frontotemporal dementia to understand how these conditions affect cells, with the goal of finding new ways to treat them.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Massachusetts Institute of Technology NIH-funded |
| Lab location | 1 site (Cambridge, United States) |
| Project ID | NIH-10989920 on NIH RePORTER |
What this research studies
This research investigates the molecular mechanisms underlying amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD) by analyzing brain samples at a single-cell level. The study aims to profile the transcriptional and epigenomic changes in patients to uncover shared and distinct genetic signatures of these diseases. By integrating various datasets, the researchers will identify potential therapeutic targets and validate their effects through advanced experimental techniques. The findings will be shared with the broader scientific community to enhance understanding and treatment options.
Who could benefit from this research
Good fit: Ideal candidates for this research are adults diagnosed with amyotrophic lateral sclerosis or frontotemporal dementia.
Not a fit: Patients with other neurodegenerative diseases not related to ALS or FTD may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to the discovery of new therapeutic targets that may improve treatment options for patients with ALS and FTD.
How similar studies have performed: Previous research has shown promise in identifying genetic and molecular targets in neurodegenerative diseases, suggesting that this approach could yield valuable insights.
Where this research is happening
Cambridge, United States
- Massachusetts Institute of Technology — Cambridge, United States (Active)
Researchers
- Principal investigator: Kellis, Manolis — Massachusetts Institute of Technology
- Study coordinator: Kellis, Manolis
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.