Creating better animal models for Alpha-1 antitrypsin deficiency
Official title: Human Genetic Disease Modeling and Physiology Core
Funded at Univ of Massachusetts Med Sch Worcester · NIH-11146477 · Data updated 2026-11-12
This work aims to develop improved animal models to help us understand and find new treatments for 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 start | August 9, 2021 |
| Project end | July 31, 2026 |
| Funding year | 2025 |
| Award (this year) | $256,865 |
| NIH project number | 5P01HL158506-05 |
| Project ID | NIH-11146477 on NIH RePORTER |
Who could benefit from this research
✓Could help
This foundational work is for patients with Alpha-1 antitrypsin deficiency who may benefit from future advanced therapies developed using these models.
✕Less likely to help
Patients seeking immediate treatment options will not directly benefit from this foundational animal model development.
What this research studies
To develop effective treatments for Alpha-1 antitrypsin deficiency, we need animal models that accurately show the same problems seen in people with the condition. This project focuses on creating and studying new mouse and ferret models that better mimic the human disease. These models will be used to test potential new therapies, including gene therapy and genome editing, and to set clear goals for how well these treatments should work. By using these improved models, we hope to speed up the discovery of successful therapies for patients.
Why it matters
Potential benefit: If successful, this work could lead to more effective gene therapies and genome editing approaches for Alpha-1 antitrypsin deficiency.
How similar studies have gone: While existing animal models have provided some insights, this work aims to create more accurate and relevant models for testing advanced therapies, representing a novel approach to improving disease modeling.
Where this research is happening
-
Univ of Massachusetts Med Sch Worcester
Worcester, United States Active
Researchers
- Principal investigator: Gruntman, Alisha Marie — Univ of Massachusetts Med Sch Worcester
- Study coordinator: Gruntman, Alisha Marie
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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.