How KIF5A gene causes RNA problems in ALS
Official title: RNA Processing Misregulation in KIF5A ALS
Funded at Monell Chemical Senses Center · NIH-11474448 · Data updated 2026-11-12
Researchers aim to find out whether mutations in the KIF5A gene change how nerve cells handle RNA in people with ALS and whether that leads to motor neuron damage.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Monell Chemical Senses Center NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project start | January 1, 2025 |
| Project end | December 31, 2028 |
| Funding year | 2026 |
| Award (this year) | $249,000 |
| NIH project number | 4R00NS140635-02 |
| Project ID | NIH-11474448 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with ALS—especially those with known KIF5A mutations or early-stage motor symptoms—would be the most relevant candidates for related sample donation or future trials.
✕Less likely to help
Patients without KIF5A-related ALS or those needing immediate clinical treatment are unlikely to receive direct, near-term benefit from this basic lab research.
What this research studies
As someone with ALS, I would be told that the team uses computer models and lab-grown spinal tissue made from human cells to see how mutated KIF5A binds to RNAs and affects cellular stress-response proteins. They will map which RNAs KIF5A prefers, study how it interacts with other ALS-linked genes, and examine changes in RNA production, localization, and RNA-binding protein interactions. The work mixes machine learning predictions with hands-on laboratory experiments to uncover molecular steps that may cause motor neuron loss.
Why it matters
Potential benefit: If successful, this work could reveal new molecular targets to prevent RNA misprocessing and protect motor neurons in ALS.
How similar studies have gone: Studies of RNA-binding proteins like TDP-43 and FUS have provided important insights in ALS, but applying this approach specifically to KIF5A is relatively new and exploratory.
Where this research is happening
-
Monell Chemical Senses Center
Philadelphia, United States Active
Researchers
- Principal investigator: Le, Phuong — Monell Chemical Senses Center
- Study coordinator: Le, Phuong
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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.