Combinatorial Therapy for SPG4 hereditary spastic paraplegia
Funded at Drexel University · NIH-11450314 · Data updated 2026-11-12
Aims to test gene-based treatments to help people with SPG4-related hereditary spastic paraplegia manage mobility and related symptoms.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Drexel University NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project start | September 15, 2026 |
| Project end | June 30, 2031 |
| Funding year | 2026 |
| Award (this year) | $619,937 |
| NIH project number | 1R01NS147575-01A1 |
| Project ID | NIH-11450314 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates are individuals with SPG4-HSP or related hereditary spastic paraplegia seeking future therapeutic options and who could participate in future clinical translation efforts.
✕Less likely to help
Patients without SPG4-HSP or those not eligible for future translation studies may not benefit.
What this research studies
This project uses mouse models of SPG4-HSP to test new gene therapy approaches aimed at preventing nerve degeneration and improving movement. A multi-PI team at Drexel University plans to combine modern techniques to create therapeutic regimens that replace mutant spastin with the healthy version and address disease mechanisms. The work focuses on translating preclinical findings into strategies that could one day help patients with SPG4-HSP maintain independence and quality of life.
Why it matters
Potential benefit: If successful, the research could lead to therapies that slow or prevent nerve degeneration and improve mobility for people with SPG4-HSP.
How similar studies have gone: Previous preclinical work in mouse models has shown promise for gene therapy approaches in SPG4-HSP, indicating potential for translation.
Where this research is happening
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Drexel University
Philadelphia, United States Active
Researchers
- Principal investigator: Baas, Peter W. — Drexel University
- Study coordinator: Baas, Peter W.
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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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.
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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.