Creating a zebrafish model to study a genetic neuromuscular disease.
Generation and characterization of a Cre-Lox regulated transgenic zebrafish model of SBMA
This study is creating a special zebrafish model to help find new treatments for spinal and bulbar muscular atrophy (SBMA), a genetic disease that mainly affects men, so researchers can quickly test potential therapies in a way that's both fast and affordable.
Quick facts
| Grant type | R03 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Philadelphia College of Osteopathic Med NIH-funded |
| Lab location | 1 site (Philadelphia, United States) |
| Project ID | NIH-10784254 on NIH RePORTER |
What this research studies
This research focuses on spinal and bulbar muscular atrophy (SBMA), a genetic neuromuscular disease primarily affecting men. The team aims to develop a zebrafish model that can mimic the disease by using a specific genetic modification technique called Cre-Lox. This model will allow for rapid testing of potential therapies and drug compounds in a cost-effective manner, providing insights that could lead to new treatments for SBMA. The zebrafish's biological similarities to humans make it an ideal candidate for this type of research.
Who could benefit from this research
Good fit: Ideal candidates for this research are adult males diagnosed with spinal and bulbar muscular atrophy.
Not a fit: Patients with other neuromuscular diseases or those not diagnosed with SBMA may not benefit from this research.
Why it matters
Potential benefit: If successful, this research could lead to the identification of new therapeutic options for patients suffering from SBMA.
How similar studies have performed: Previous research using zebrafish models for other genetic diseases has shown promising results, indicating that this approach could be effective for SBMA as well.
Where this research is happening
Philadelphia, United States
- Philadelphia College of Osteopathic Med — Philadelphia, United States (Active)
Researchers
- Principal investigator: Montie, Heather L — Philadelphia College of Osteopathic Med
- Study coordinator: Montie, Heather L
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.