Understanding axon degeneration in hereditary spastic paraplegia and ALS using patient-derived neurons
Official title: Mechanisms and rescue of axonal degeneration in hereditary spastic paraplegia neurons
Funded at University of Illinois at Chicago · NIH-11528579 · Data updated 2026-11-12
Aimed at understanding why certain nerve fibers die in hereditary spastic paraplegia and ALS by studying neurons made from patients’ own stem cells.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Illinois at Chicago NIH-funded |
| Lab location | 1 site (Chicago, UNITED STATES) |
| Project start | May 1, 2026 |
| Project end | April 30, 2027 |
| Funding year | 2026 |
| Award (this year) | $400,825 |
| NIH project number | 1R56NS142274-01A1 |
| Project ID | NIH-11528579 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients with hereditary spastic paraplegia or ALS, or cells derived from them, may contribute iPSC models for study.
✕Less likely to help
Individuals without HSP/ALS or without access to patient-derived iPSCs may not directly benefit from this research.
What this research studies
Researchers will use neurons created from patients’ induced pluripotent stem cells to study how spatacsin and spastizin affect autophagy, lysosome transport, and mitochondrial health. The team will look at how these processes interact to cause axon degeneration and test ideas for rescuing nerve damage. The work builds on prior findings of impaired mitochondrial dynamics and altered autophagy in patient-derived neurons. Results could point to new targets to protect nerves in people with HSP or ALS.
Why it matters
Potential benefit: Findings could lead to new strategies to prevent or slow nerve degeneration in HSP and ALS.
How similar studies have gone: Previous studies with patient-derived neurons have shown relevant disease mechanisms; this project extends those findings to axonal transport and autophagy.
Where this research is happening
-
University of Illinois at Chicago
Chicago, United States Active
Researchers
- Principal investigator: Li, Xue-Jun — University of Illinois at Chicago
- Study coordinator: Li, Xue-Jun
Related research
Other NIH-funded projects on the same topics.
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Combinatorial Therapy for SPG4 hereditary spastic paraplegia
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Understanding how full-length spastin works and its role in hereditary spastic paraplegia
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Finding hidden genetic causes of hereditary spastic paraplegia
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A new antibody-based therapy for SPG4 hereditary spastic paraplegia
A small NIH grant is funding a therapy aimed at reducing mutant M1 spastin protein to help people with SPG4-HSP, a gradual leg-weakening neurodegenerative condition.
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Understanding nerve damage in hereditary spastic paraplegia
Researchers are using genetically modified rats and patient-derived stem cells to learn how gene mutations cause nerve fiber damage in people with hereditary spastic paraplegia.
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Understanding and treating a genetic condition that affects movement in children
This study is looking at how certain genes affect movement problems in children with hereditary spastic paraplegia and is testing new treatments that might help improve their nerve function and overall health.
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.