Investigating how DNA damage and repair mechanisms are altered in ALS and frontotemporal dementia.
Defining the altered FUS-PARP-1-DNA Ligase III axis and its implications to nuclear and mitochondrial genome damage response in Amyotrophic Lateral Sclerosis (ALS) and Frontotemporal Dementia (FTD)
This study is looking at how problems with DNA repair might contribute to conditions like ALS and frontotemporal dementia, and it’s testing ways to fix these issues in cells from patients to help develop new treatments.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Methodist Hospital Research Institute NIH-funded |
| Lab location | 1 site (Houston, United States) |
| Project ID | NIH-9980670 on NIH RePORTER |
What this research studies
This research focuses on understanding the mechanisms behind DNA damage and defective repair in patients with amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). It examines the role of the FUS protein in protecting the genome and how mutations in this protein lead to impaired DNA repair processes. By utilizing advanced techniques like CRISPR/Cas9, the study aims to correct these mutations in patient-derived cells, potentially restoring normal DNA repair functions. The findings could pave the way for new therapeutic strategies targeting genome repair in neurodegenerative diseases.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with ALS or frontotemporal dementia, particularly those with known mutations in the FUS gene.
Not a fit: Patients without a diagnosis of ALS or frontotemporal dementia, or those with different underlying genetic causes of neurodegeneration, may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to innovative treatments that improve DNA repair mechanisms in patients with ALS and FTD, potentially slowing disease progression.
How similar studies have performed: Previous research has shown promising results in understanding DNA repair mechanisms in neurodegenerative diseases, indicating that this approach may yield valuable insights.
Where this research is happening
Houston, United States
- Methodist Hospital Research Institute — Houston, United States (Active)
Researchers
- Principal investigator: Hegde, Muralidhar L — Methodist Hospital Research Institute
- Study coordinator: Hegde, Muralidhar L
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.