Noninvasive gene delivery to the brain and spinal cord
Bioengineering of highly effective AAV vectors for noninvasive gene delivery to the nervous system
['FUNDING_R01'] · TEMPLE UNIV OF THE COMMONWEALTH · NIH-11308642
Using newly engineered AAV9 gene carriers given through a simple IV to reach brain and spinal cord cells, aiming to help people with spinal cord injuries and other nervous system disorders.
Quick facts
| Phase | ['FUNDING_R01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | TEMPLE UNIV OF THE COMMONWEALTH (nih funded) |
| Locations | 1 site (PHILADELPHIA, UNITED STATES) |
| Trial ID | NIH-11308642 on ClinicalTrials.gov |
What this research studies
Researchers are engineering improved AAV9 viral carriers that can cross the blood-brain barrier after a single intravenous injection to reach many types of brain and spinal cord cells. In the lab they will test how well these vectors deliver marker genes to neurons, astrocytes, oligodendrocytes, and microglia across several strains of adult mice. They will then use the best vectors to deliver therapeutic genes that target neuronal growth programs (for example, let-7 miRNA modulation) to see if injured spinal cords regrow axons and recover function. The work is preclinical and done in animal models to validate a noninvasive delivery method that could later be adapted for human neurological conditions.
Who could benefit from this research
Good fit: People with spinal cord injury or other CNS disorders might be future candidates for therapies that use this delivery method once human trials begin.
Not a fit: Patients should not expect direct benefit now because the work is preclinical in animals and does not offer current patient enrollment or treatment.
Why it matters
Potential benefit: If successful, this could enable safer, noninvasive gene therapies that reach many cell types in the CNS and improve recovery after spinal cord injury and other neurological diseases.
How similar studies have performed: Engineered AAV vectors have shown promising results in animal studies for crossing the blood-brain barrier, but translating that success to humans remains unproven.
Where this research is happening
PHILADELPHIA, UNITED STATES
- TEMPLE UNIV OF THE COMMONWEALTH — PHILADELPHIA, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: LI, SHUXIN — TEMPLE UNIV OF THE COMMONWEALTH
- Study coordinator: LI, SHUXIN
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.
Conditions: Alzheimer disease dementia, Alzheimer syndrome, Alzheimer's Disease