Investigating the role of tRNA in ALS and frontotemporal dementia
The tRNA pool in C9-ALS/FTD
This study is looking into how certain genetic changes linked to ALS and frontotemporal dementia affect the way our cells make proteins, with the hope of finding new ways to help people living with these conditions.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Thomas Jefferson University NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project ID | NIH-10817770 on NIH RePORTER |
What this research studies
This research focuses on understanding the mechanisms behind C9orf72-mediated amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), two severe neurodegenerative diseases. The study aims to explore how the cellular tRNA pool, which is essential for protein synthesis, responds to the unique challenges posed by the genetic mutations associated with these conditions. By examining the translation of specific nucleotide sequences that lead to the production of harmful proteins, the research seeks to identify potential therapeutic targets that could improve treatment options for affected patients.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with C9orf72-mediated ALS or FTD.
Not a fit: Patients with other forms of ALS or FTD that do not involve the C9orf72 genetic mutation may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to new therapeutic strategies for treating ALS and FTD, potentially improving patient outcomes.
How similar studies have performed: Previous research has shown promising results in understanding the genetic mechanisms of ALS and FTD, suggesting that this approach could yield valuable insights.
Where this research is happening
Philadelphia, United States
- Thomas Jefferson University — Philadelphia, United States (Active)
Researchers
- Principal investigator: Hou, Ya-Ming — Thomas Jefferson University
- Study coordinator: Hou, Ya-Ming
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.