Finding better ways to deliver gene therapy for liver diseases.
Exploitation of the natural biodiversity of AAV to identify “super transducers"
['FUNDING_P01'] · UNIV OF MASSACHUSETTS MED SCH WORCESTER · NIH-10914224
This study is working on making gene therapy better for people with alpha-1-antitrypsin deficiency by finding new ways to deliver the treatment to the liver, which could lead to improved health and liver function for patients.
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-10914224 on ClinicalTrials.gov |
What this research studies
This research focuses on improving gene therapy for alpha-1-antitrypsin deficiency (AATD) by developing new adeno-associated virus (AAV) capsids that enhance gene delivery to the liver. The team will utilize advanced sequencing techniques to identify novel AAV variants from human and non-human primate tissues. By analyzing these variants, the research aims to create more effective gene delivery systems that can overcome existing challenges, such as pre-existing immunity to AAV. Patients may benefit from more efficient treatments that could improve their liver function and overall health.
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 enhanced gene therapy options.
Not a fit: Patients with liver diseases not related to 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 gene therapies for patients with liver diseases, particularly those with AAT deficiency.
How similar studies have performed: Previous research has shown promise in using AAV vectors for gene therapy, but this approach aims to innovate by identifying new capsid variants, making it a novel exploration.
Where this research is happening
WORCESTER, UNITED STATES
- UNIV OF MASSACHUSETTS MED SCH WORCESTER — WORCESTER, UNITED STATES (ACTIVE)
Researchers
- Principal investigator: WANG, DAN — UNIV OF MASSACHUSETTS MED SCH WORCESTER
- Study coordinator: WANG, DAN
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.