Investigating protein balance in a model of frontotemporal dementia
Protein homeostasis in a frontotemporal dementia iPSC model
This study is looking at how changes in a specific gene can impact the health of brain cells, which is important for understanding diseases like frontotemporal dementia, and it aims to find new ways to help patients with these conditions.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Pittsburgh at Pittsburgh NIH-funded |
| Lab location | 1 site (Pittsburgh, United States) |
| Project ID | NIH-10525437 on NIH RePORTER |
What this research studies
This research focuses on understanding how mutations in the VCP gene affect protein homeostasis in neurons, which is crucial for their health and function. By creating human neuron models with specific mutations, researchers aim to explore the mechanisms behind neurodegenerative diseases, particularly frontotemporal dementia. The study will compare the effects of a mutation linked to familial frontotemporal dementia against normal protein function, providing insights into cellular stress responses and potential therapeutic targets. Patients may benefit from advancements in understanding and treating related neurodegenerative conditions.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals with a family history of frontotemporal dementia or those diagnosed with related neurodegenerative conditions.
Not a fit: Patients with neurodegenerative diseases not linked to VCP mutations may not receive direct benefits from this research.
Why it matters
Potential benefit: If successful, this research could lead to new strategies for treating frontotemporal dementia and related neurodegenerative diseases.
How similar studies have performed: Previous research has shown promising results in understanding protein homeostasis in neurodegenerative diseases, suggesting that this approach could yield valuable insights.
Where this research is happening
Pittsburgh, United States
- University of Pittsburgh at Pittsburgh — Pittsburgh, United States (Active)
Researchers
- Principal investigator: Chu, Charleen T — University of Pittsburgh at Pittsburgh
- Study coordinator: Chu, Charleen T
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.