Investigating gene therapy for Prader-Willi syndrome
Prader-Willi syndrome (PWS) gene-domain and AAV miniaturization for gene therapy
This study is exploring a new gene therapy for Prader-Willi syndrome that aims to replace missing genes using a tiny virus, which could help improve treatments for people living with this condition.
Quick facts
| Grant type | R21 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Pittsburgh at Pittsburgh NIH-funded |
| Lab location | 1 site (Pittsburgh, United States) |
| Project ID | NIH-10771280 on NIH RePORTER |
What this research studies
This research focuses on developing a new gene therapy approach for Prader-Willi syndrome (PWS), a complex genetic disorder. The project aims to miniaturize key genetic elements into a single adeno-associated virus (AAV) vector to potentially replace missing or silent genes associated with PWS. By testing the expression of these miniaturized genes in cells, the researchers hope to better understand how gene loss affects the symptoms of PWS. This innovative method could lead to more effective treatments for the condition.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with Prader-Willi syndrome who may benefit from advanced genetic therapies.
Not a fit: Patients with other genetic disorders or those without a diagnosis of Prader-Willi syndrome may not receive any benefit from this research.
Why it matters
Potential benefit: If successful, this research could provide a novel gene therapy that significantly improves the quality of life for individuals with Prader-Willi syndrome.
How similar studies have performed: While gene therapy for genetic disorders is a growing field, this specific approach for Prader-Willi syndrome is novel and has not been extensively tested in prior studies.
Where this research is happening
Pittsburgh, United States
- University of Pittsburgh at Pittsburgh — Pittsburgh, United States (Active)
Researchers
- Principal investigator: Nicholls, Robert D — University of Pittsburgh at Pittsburgh
- Study coordinator: Nicholls, Robert D
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.