Finding better gene delivery methods for Alpha-1 Antitrypsin Deficiency
Exploitation of the natural biodiversity of AAV to identify “super transducers"
This project aims to discover new and more effective ways to deliver gene therapy to the liver for people with alpha-1 antitrypsin deficiency.
Quick facts
| Grant type | P01 program project |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Univ of Massachusetts Med Sch Worcester NIH-funded |
| Lab location | 1 site (Worcester, United States) |
| Project ID | NIH-11146484 on NIH RePORTER |
What this research studies
For individuals with alpha-1 antitrypsin deficiency, delivering gene therapy to the liver has faced challenges, especially when the body's immune system already recognizes the delivery method. This work focuses on finding improved adeno-associated virus (AAV) capsids, which are like the 'shells' that carry genetic material into cells. Researchers are exploring a wide variety of natural AAV forms found in both human and non-human primate tissues to identify those that are exceptionally good at reaching liver cells. The goal is to create a collection of these 'super transducer' AAV capsids that can deliver genes more efficiently and avoid immune responses, paving the way for better gene therapies.
Who could benefit from this research
Good fit: Patients with alpha-1 antitrypsin deficiency, particularly those who might benefit from liver-directed gene therapy, are the focus of this foundational research.
Not a fit: Patients whose conditions are not related to liver-directed gene therapy or alpha-1 antitrypsin deficiency may not directly benefit from this specific research.
Why it matters
Potential benefit: If successful, this work could lead to more effective and safer gene therapies for alpha-1 antitrypsin deficiency and potentially other liver-related genetic conditions.
How similar studies have performed: While the specific 'super transducer' capsids are novel, the general approach of using AAV for gene delivery has shown promise in other studies, and new AAV variants have been previously identified.
Where this research is happening
Worcester, United States
- Univ of Massachusetts Med Sch Worcester — Worcester, United States (Active)
Researchers
- Principal investigator: Wang, Dan — Univ of Massachusetts Med Sch Worcester
- Study coordinator: Wang, Dan
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.