Evaluating a gene therapy for neuromuscular junction diseases.
Official title: EFFICACY AND SAFETY EVALUATION OF LEAD GENE THERAPY CANDIDATE IN NEUROMUSCULAR JUNCTION (NMJ) DISEASE MOUSE MODELS AND BIOANALYTICAL ASSAY METHODS DEVELOPMENT
Funded at Lovelace Biomedical Research Institute · NIH-10954013 · Data updated 2026-11-12
This study is exploring a new gene therapy using special viruses to help treat Congenital Myasthenic Syndromes in mice, with the goal of finding safe and effective ways to improve muscle function for people with this condition.
Quick facts
| Grant type | NIH-funded research |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Lovelace Biomedical Research Institute NIH-funded |
| Lab location | 1 site (Albuquerque, United States) |
| Project start | September 29, 2023 |
| Project end | September 28, 2026 |
| Funding year | 2023 |
| Award (this year) | $1,595,870 |
| NIH project number | 75N95019D00012-0-759502300002-1 |
| Project ID | NIH-10954013 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal candidates for this research are individuals diagnosed with Congenital Myasthenic Syndromes or related neuromuscular junction disorders.
✕Less likely to help
Patients with other types of neuromuscular disorders that are not related to congenital myasthenia may not benefit from this research.
What this research studies
This research focuses on testing a new gene therapy approach using adeno-associated viruses to treat Congenital Myasthenic Syndromes (CMS) in mouse models. The study aims to assess both the safety and effectiveness of these gene therapy candidates, as well as develop methods to analyze how the therapy spreads in the body and its effects on muscle function. By using advanced bioanalytical techniques, researchers hope to gather important data that could lead to new treatment options for patients with neuromuscular junction diseases.
Why it matters
Potential benefit: If successful, this research could lead to innovative gene therapies that significantly improve the quality of life for patients with Congenital Myasthenic Syndromes.
How similar studies have gone: Previous research has shown promise in using gene therapy for neuromuscular diseases, indicating that this approach could be a viable treatment option.
Where this research is happening
-
Lovelace Biomedical Research Institute
Albuquerque, United States Active
Researchers
- Principal investigator: Robertson, Mike — Lovelace Biomedical Research Institute
- Study coordinator: Robertson, Mike
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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.