Understanding nerve damage in hereditary spastic paraplegia
Official title: Mechanisms Underlying Axonopathy in Hereditary Spastic Paraplegia
Funded at University of Wisconsin-Madison · NIH-11061852 · Data updated 2026-11-12
This study is looking into how genetic changes cause nerve damage in hereditary spastic paraplegia (HSP), which affects balance and muscle strength, with the hope of finding new treatments to help people with this condition.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Wisconsin-Madison NIH-funded |
| Lab location | 1 site (Madison, United States) |
| Project start | May 1, 2022 |
| Project end | March 31, 2027 |
| Funding year | 2025 |
| Award (this year) | $375,430 |
| NIH project number | 5R01NS124165-04 |
| Project ID | NIH-11061852 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with hereditary spastic paraplegia or those exhibiting symptoms related to this condition.
✕Less likely to help
Patients with other neurological disorders unrelated to hereditary spastic paraplegia may not benefit from this research.
What this research studies
This research investigates the mechanisms behind nerve damage in hereditary spastic paraplegia (HSP), a condition that leads to balance issues, muscle weakness, and potentially paralysis. By using animal models and induced pluripotent stem cells, the team aims to uncover how genetic mutations contribute to neurodegeneration. They employ advanced techniques like CRISPR for genome editing to create models that mimic the disease, allowing for a detailed study of disease progression and potential therapeutic targets. The ultimate goal is to identify new treatments that could improve patient outcomes.
Why it matters
Potential benefit: If successful, this research could lead to new therapies that significantly improve mobility and quality of life for patients with hereditary spastic paraplegia.
How similar studies have gone: Previous research has shown promise in understanding similar neurodegenerative conditions, suggesting that this approach may yield valuable insights.
Where this research is happening
-
University of Wisconsin-Madison
Madison, United States Active
Researchers
- Principal investigator: Audhya, Anjon — University of Wisconsin-Madison
- Study coordinator: Audhya, Anjon
Related research
Other NIH-funded projects on the same topics.
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Understanding axon degeneration in hereditary spastic paraplegia and ALS using patient-derived neurons
Aimed at understanding why certain nerve fibers die in hereditary spastic paraplegia and ALS by studying neurons made from patients’ own stem cells.
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Combinatorial Therapy for SPG4 hereditary spastic paraplegia
Aims to test gene-based treatments to help people with SPG4-related hereditary spastic paraplegia manage mobility and related symptoms.
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Understanding how full-length spastin works and its role in hereditary spastic paraplegia
This grant aims to uncover how the spastin protein uses ATP to cut and regrow microtubules, shedding light on the most common inherited cause of a neurodegenerative leg-weakness disorder.
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Finding hidden genetic causes of hereditary spastic paraplegia
Using whole genome sequencing to find genetic causes for people with hereditary spastic paraplegia whose earlier genetic tests came up inconclusive.
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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.
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.