How AAV gene therapy targets different organs
Determinants of AAV Tropism
Looking for ways to make AAV gene therapy reach the right organs at lower doses for people who need genetic treatments.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Duke University NIH-funded |
| Lab location | 1 site (Durham, United States) |
| Project ID | NIH-11322047 on NIH RePORTER |
What this research studies
I'm told the researchers want to learn why different AAV virus shells go to certain organs and how to make them work at lower doses. They create large libraries of engineered AAV capsids and cycle them through mice, pigs, and macaques to find variants that work across species. The team will study capsid structure, where vectors travel in the body, and the molecular steps that improve cell entry and expression. The goal is to pick AAVs that could allow effective gene delivery with less risk to liver and kidneys in future human use.
Who could benefit from this research
Good fit: People with genetic disorders who are current or future candidates for AAV-delivered gene therapy would be the most relevant candidates.
Not a fit: Patients whose conditions are not treatable with AAV-delivered genes or who are ineligible for gene therapy procedures would be unlikely to benefit directly from this research.
Why it matters
Potential benefit: Could enable effective AAV gene therapy at lower doses, reducing liver and kidney toxicity and improving safety for patients.
How similar studies have performed: Preclinical work has produced potent cross-species AAV variants and shown promise, but clinical experience has been mixed because of dose-related toxicities, so safe human translation remains a challenge.
Where this research is happening
Durham, United States
- Duke University — Durham, United States (Active)
Researchers
- Principal investigator: Asokan, Aravind — Duke University
- Study coordinator: Asokan, Aravind
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.