Controlled PTEN lowering to help spinal cord nerves regrow
Regulated PTEN expression to overcome barriers to corticospinal tract regeneration after spinal cord injury
This project uses a tunable gene therapy approach to temporarily reduce a growth-blocking protein called PTEN and clear scar molecules to encourage nerve fibers in the spinal cord to regrow and improve movement in adults with spinal cord injury.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Louisville NIH-funded |
| Lab location | 1 site (Louisville, United States) |
| Project ID | NIH-11177008 on NIH RePORTER |
What this research studies
From a patient's view, researchers will deliver controllable gene therapy using AAV vectors to lower PTEN in injured nerve cells for defined times, and they will use an enzyme to break down scar-like molecules that block regrowth. They will test different timing and combinations to promote corticospinal tract axon regrowth while avoiding harmful long-term effects seen with permanent PTEN removal. The team will measure anatomical nerve regrowth, functional recovery of voluntary movement, and signs of late pathologies in preclinical adult models of spinal cord injury. Findings will guide safer approaches that could be moved toward human testing if successful.
Who could benefit from this research
Good fit: The eventual candidates for therapies developed from this work would be adults (21+) with spinal cord injuries affecting voluntary movement pathways like the corticospinal tract.
Not a fit: People whose paralysis is not due to corticospinal tract injury, those with contraindications to viral gene therapy, or those needing different treatment approaches may not receive direct benefit from this specific line of work.
Why it matters
Potential benefit: If successful, this work could lead to therapies that restore voluntary movement after spinal cord injury by promoting targeted nerve regrowth while reducing long-term side effects.
How similar studies have performed: Prior animal studies showed that permanent PTEN deletion can improve recovery but produced later pathologies, so this project builds on promising results by testing temporary control and scar degradation to improve safety and durability.
Where this research is happening
Louisville, United States
- University of Louisville — Louisville, United States (Active)
Researchers
- Principal investigator: Metcalfe, Mariajose — University of Louisville
- Study coordinator: Metcalfe, Mariajose
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 below.