Creating animal models to test therapies for Alpha-1 antitrypsin deficiency
Human Genetic Disease Modeling and Physiology Core
This study is creating special mice and ferrets to help test new treatments for Alpha-1 antitrypsin deficiency, which could lead to better options for people living with this condition.
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-10914220 on NIH RePORTER |
What this research studies
This research focuses on developing animal models that mimic the conditions of patients with Alpha-1 antitrypsin deficiency, a genetic disorder affecting the lungs and liver. By creating specific mouse and ferret models, researchers aim to test new therapies, including gene therapy and genome editing, to find effective treatments. The models will help in understanding the disease better and in evaluating the success of potential therapies in a controlled environment. Patients may benefit from advancements in treatment options that arise from these studies.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Alpha-1 antitrypsin deficiency who may benefit from new therapeutic approaches.
Not a fit: Patients with other unrelated genetic disorders or those without Alpha-1 antitrypsin deficiency may not receive any benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to more effective therapies for patients suffering from Alpha-1 antitrypsin deficiency.
How similar studies have performed: Previous research has shown success in developing animal models for genetic diseases, indicating that this approach has potential for meaningful advancements.
Where this research is happening
Worcester, United States
- 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
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.