New Gene Therapies for AAT Deficiency
Models and Gene Therapies for AAT Deficiency
['FUNDING_P01'] · UNIV OF MASSACHUSETTS MED SCH WORCESTER · NIH-11146472
This program explores new gene therapy approaches to help people with Alpha-1 Antitrypsin (AAT) Deficiency.
Quick facts
| Phase | ['FUNDING_P01'] |
|---|---|
| Study type | Nih_funding |
| Sex | All |
| Sponsor | UNIV OF MASSACHUSETTS MED SCH WORCESTER (nih funded) |
| Locations | 1 site (WORCESTER, UNITED STATES) |
| Trial ID | NIH-11146472 on ClinicalTrials.gov |
What this research studies
Our program is dedicated to understanding and developing advanced treatments for Alpha-1 Antitrypsin (AAT) Deficiency, a genetic condition affecting the lungs and liver. We are working on creating better models of the disease to test new ideas and developing gene therapies that could correct the underlying genetic problem. These therapies aim to deliver healthy genes into the body, potentially restoring the missing protein and preventing disease progression. Our goal is to bring these promising new treatments closer to patients.
Who could benefit from this research
Good fit: Patients with Alpha-1 Antitrypsin Deficiency, particularly those interested in future gene therapy options, would be ideal candidates to follow this research.
Not a fit: Patients without Alpha-1 Antitrypsin Deficiency would not directly benefit from this specific gene therapy research.
Why it matters
Potential benefit: If successful, this research could lead to long-lasting gene therapies that directly address the cause of AAT Deficiency, potentially improving lung and liver health for patients.
How similar studies have performed: Gene therapy for other genetic disorders has shown promise, and ongoing research in AAT Deficiency is actively exploring similar genetic approaches.
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.