Investigating a new target for treating frontal temporal dementia
Official title: Implicating the major G-quadruplex helicase, DHX36, as a novel pathogenic modifier of C9ORF72 frontal temporal dementia
Funded at Ball State University · NIH-10974535 · Data updated 2026-11-12
This study is looking into how a gene mutation related to frontotemporal dementia affects brain cells, and it’s exploring a special enzyme that might help reduce harmful proteins, with the hope of finding new treatments for people living with this condition.
Quick facts
| Grant type | R15 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Ball State University NIH-funded |
| Lab location | 1 site (Muncie, United States) |
| Project start | May 1, 2020 |
| Project end | July 31, 2027 |
| Funding year | 2024 |
| Award (this year) | $475,516 |
| NIH project number | 2R15AG067291-02 |
| Project ID | NIH-10974535 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research include individuals diagnosed with frontal temporal dementia or those carrying the C9ORF72 mutation.
✕Less likely to help
Patients with other forms of dementia unrelated to the C9ORF72 mutation may not benefit from this research.
What this research studies
This research focuses on understanding the molecular mechanisms behind frontal temporal dementia (FTD), particularly how a mutation in the C9ORF72 gene contributes to the disease. The study explores the role of a specific enzyme, DHX36, which unwinds harmful DNA and RNA structures known as G-quadruplexes that are linked to neuron degeneration. By examining how DHX36 affects the production of toxic proteins in stressed cells, the researchers aim to develop a novel treatment strategy for FTD. This innovative approach could lead to new therapeutic options for patients suffering from this debilitating condition.
Why it matters
Potential benefit: If successful, this research could lead to new treatments that slow or prevent the progression of frontal temporal dementia.
How similar studies have gone: While the specific approach of targeting DHX36 in FTD is novel, similar strategies targeting G-quadruplexes have shown promise in other neurological conditions.
Where this research is happening
-
Ball State University
Muncie, United States Active
Researchers
- Principal investigator: Smaldino, Philip J — Ball State University
- Study coordinator: Smaldino, Philip J
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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.