Investigating how AAV capsid and promoter interactions affect gene expression in the brain
AAV capsid-promoter interactions determines CNS cell selective gene expression in vivo
This study is looking at how changing certain virus parts can help deliver genes more effectively to specific brain cells, which could make gene therapies better for treating conditions like spinal muscular atrophy.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Univ of North Carolina Chapel Hill NIH-funded |
| Lab location | 1 site (Chapel Hill, United States) |
| Project ID | NIH-10992114 on NIH RePORTER |
What this research studies
This research explores how specific interactions between adeno-associated virus (AAV) capsids and gene promoters can influence which types of brain cells express certain genes. By modifying the AAV capsid and using different promoters, the researchers aim to achieve targeted gene expression in the central nervous system (CNS). This approach could enhance the effectiveness of gene therapies for genetic disorders affecting the CNS, such as spinal muscular atrophy. The study involves both laboratory experiments and potential clinical applications to improve gene delivery methods.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals with genetic disorders that affect the central nervous system.
Not a fit: Patients with non-genetic CNS disorders or those not affected by genetic conditions may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to more effective gene therapies for patients with central nervous system disorders.
How similar studies have performed: Previous research has shown promising results with AAV vectors in CNS clinical trials, indicating that this approach has potential for success.
Where this research is happening
Chapel Hill, United States
- Univ of North Carolina Chapel Hill — Chapel Hill, United States (Active)
Researchers
- Principal investigator: Mccown, Thomas J. — Univ of North Carolina Chapel Hill
- Study coordinator: Mccown, Thomas J.
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.