Improving gene therapy for Alpha-1 antitrypsin deficiency
Optimized Gene Replacement for AAT deficiency and Modeling of Clinical Outcomes in small and large animal models
This study is exploring new gene therapy treatments for Alpha-1 antitrypsin deficiency, a condition that impacts lung health, by using advanced technology to create animal models that help us understand how to better restore normal protein levels and improve care for patients like you.
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-10914222 on NIH RePORTER |
What this research studies
This research focuses on developing optimized gene replacement therapies for Alpha-1 antitrypsin deficiency (AATD), a genetic disorder that affects lung function. The team is utilizing advanced CRISPR technology to create animal models that mimic the condition, allowing for better understanding and testing of potential treatments. By investigating the effectiveness of gene therapy in restoring normal protein levels, the research aims to provide insights into how to improve current treatment options for patients. The approach includes both small and large animal models to ensure comprehensive evaluation of clinical outcomes.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Alpha-1 antitrypsin deficiency, particularly those experiencing lung-related symptoms.
Not a fit: Patients without Alpha-1 antitrypsin deficiency or those who do not exhibit symptoms related to lung function may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to more effective gene therapies that significantly improve lung function and quality of life for patients with AAT deficiency.
How similar studies have performed: Previous research using gene therapy for AAT deficiency has shown some promise, but this approach aims to enhance the efficacy of those methods.
Where this research is happening
Worcester, United States
- Univ of Massachusetts Med Sch Worcester — Worcester, United States (Active)
Researchers
- Principal investigator: Flotte, Terence R. — Univ of Massachusetts Med Sch Worcester
- Study coordinator: Flotte, Terence R.
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.