Gene therapies for treating AAT deficiency
Models and Gene Therapies for AAT Deficiency
['FUNDING_P01'] · UNIV OF MASSACHUSETTS MED SCH WORCESTER · NIH-10914216
This study is exploring new treatments for people with Alpha-1 Antitrypsin deficiency, a genetic condition that can affect your lungs and liver, and it may offer you a chance to join clinical trials testing these exciting new therapies.
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-10914216 on ClinicalTrials.gov |
What this research studies
This research focuses on developing models and gene therapies specifically for Alpha-1 Antitrypsin (AAT) deficiency, a genetic condition that can lead to serious lung and liver diseases. The approach involves creating a structured program that includes financial management and communication among various research components. By utilizing advanced genetic interventions, the research aims to improve treatment options for patients suffering from this deficiency. Patients may have the opportunity to participate in clinical trials that test these innovative therapies.
Who could benefit from this research
Good fit: Ideal candidates for participation are individuals diagnosed with Alpha-1 Antitrypsin deficiency who are seeking new treatment options.
Not a fit: Patients who do not have AAT deficiency or those with advanced liver or lung disease may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide new gene therapy options that significantly improve the health and quality of life for patients with AAT deficiency.
How similar studies have performed: Previous research in gene therapies for genetic disorders has shown promising results, indicating potential for success in this area as well.
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.