Developing gene therapy to treat limb girdle muscular dystrophy type 2A
Optimizing and validation of gene therapy vectors to treat limb girdle muscular dystophy
This study is working on a new gene therapy for people with limb girdle muscular dystrophy type 2A (LGMD2A) to help improve muscle function and quality of life by safely delivering a key gene that supports muscle health.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California Los Angeles NIH-funded |
| Lab location | 1 site (Los Angeles, United States) |
| Project ID | NIH-10837773 on NIH RePORTER |
What this research studies
This research focuses on creating a gene therapy specifically for limb girdle muscular dystrophy type 2A (LGMD2A), a genetic disorder that leads to muscle wasting. The approach involves optimizing gene therapy vectors to ensure safe and effective delivery of the CAPN3 gene, which is crucial for muscle function. Researchers will carefully consider the unique challenges of this condition, such as avoiding heart toxicity and ensuring the therapy targets the appropriate muscle fibers. The goal is to develop a treatment that can be administered safely to patients, potentially improving their quality of life.
Who could benefit from this research
Good fit: Ideal candidates for this research are individuals diagnosed with limb girdle muscular dystrophy type 2A who are experiencing muscle weakness and mobility issues.
Not a fit: Patients with other forms of muscular dystrophy or those who do not have LGMD2A may not benefit from this specific research.
Why it matters
Potential benefit: If successful, this research could provide a groundbreaking treatment option for patients suffering from LGMD2A, potentially halting or reversing muscle degeneration.
How similar studies have performed: While there have been successful pre-clinical studies using gene therapy for similar conditions, this specific approach for LGMD2A is novel and has not yet been tested in humans.
Where this research is happening
Los Angeles, United States
- University of California Los Angeles — Los Angeles, United States (Active)
Researchers
- Principal investigator: Spencer, Melissa Jan — University of California Los Angeles
- Study coordinator: Spencer, Melissa Jan
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.