Creating a zebrafish model to study a genetic neuromuscular disease.
Official title: Generation and characterization of a Cre-Lox regulated transgenic zebrafish model of SBMA
Funded at Philadelphia College of Osteopathic Med · NIH-10784254 · Data updated 2026-11-12
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 start | September 11, 2023 |
| Project end | August 31, 2025 |
| Funding year | 2023 |
| Award (this year) | $151,600 |
| NIH project number | 1R03NS135339-01 |
| Project ID | NIH-10784254 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are adult males diagnosed with spinal and bulbar muscular atrophy.
✕Less likely to help
Patients with other neuromuscular diseases or those not diagnosed with SBMA may not benefit from this research.
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.
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 gone: 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 College of Osteopathic Med
Philadelphia, United States Active
Researchers
- Principal investigator: Montie, Heather L — Philadelphia College of Osteopathic Med
- Study coordinator: Montie, Heather L
Related research
Other NIH-funded projects on the same topics.
-
Adapting and testing a behavioral program to prevent FASD and adverse infant outcomes
A behavioral program is being tailored and tested to help pregnant and breastfeeding women reduce alcohol and other substance use to protect babies in South Africa.
-
Adapting a Behavioral Program to Prevent FASD and Harmful Infant Outcomes in SA
A program is being adapted and tested to help pregnant and breastfeeding women in South Africa reduce alcohol, tobacco, and cannabis use to prevent fetal alcohol spectrum disorder and other infant problems.
-
CD38 and muscle energy in spinal and bulbar muscular atrophy
The team is trying to lower CD38 to boost muscle energy and help men with spinal and bulbar muscular atrophy (SBMA).
-
Investigating CD38 as a potential treatment target for spinal and bulbar muscular atrophy
This study is looking at a condition called spinal and bulbar muscular atrophy (SBMA) that mainly affects men, and it aims to find out how a specific enzyme called CD38 might help improve energy in muscle cells, which could lead to new treatments for people with this condition.
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.