Controlling the dose of a viral vector used in gene therapy
Dose control of recombinant Adeno-associated virus with Chemical Epigenetic Modifiers
This study is looking at a way to improve gene therapies by using special molecules to help control how much genetic material is delivered by a virus, making treatments safer and more effective for people who need them.
Quick facts
| Grant type | Fellowship 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-10901141 on NIH RePORTER |
What this research studies
This research investigates how to better control the amount of genetic material delivered by a viral vector known as recombinant adeno-associated virus (rAAV), which is used in gene therapies. The approach involves using small molecules called Chemical Epigenetic Modifiers (CEMs) to regulate gene expression at the epigenetic level. By applying these CEMs, researchers aim to fine-tune the dosage of rAAV in human cell lines, which could lead to more effective and safer gene therapies. The study will also explore the underlying mechanisms of how these modifications affect gene expression.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals with conditions that could benefit from gene therapy, particularly those involved in ongoing clinical trials using rAAV.
Not a fit: Patients who do not have conditions treatable by gene therapy or who are not involved in relevant clinical trials may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to more precise and effective gene therapies with controlled dosing, improving patient outcomes.
How similar studies have performed: While the use of rAAV in gene therapy is established, the specific approach of using CEMs for dose control is novel and has not been widely tested.
Where this research is happening
Chapel Hill, United States
- Univ of North Carolina Chapel Hill — Chapel Hill, United States (Active)
Researchers
- Principal investigator: Wasserman, Sara Rose — Univ of North Carolina Chapel Hill
- Study coordinator: Wasserman, Sara Rose
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.