Laminin protein treatment for LAMA2 (MDC1A) congenital muscular dystrophy
Official title: Laminin protein therapy for the treatment of Laminin-alpha2 deficient congenital muscular dystrophy
Funded at University of Nevada Reno · NIH-11314568 · Data updated 2026-11-12
This project aims to develop a laminin protein therapy to slow muscle weakness in people with LAMA2 (MDC1A) congenital muscular dystrophy.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of Nevada Reno NIH-funded |
| Lab location | 1 site (Reno, United States) |
| Project start | April 1, 2024 |
| Project end | March 31, 2029 |
| Funding year | 2026 |
| Award (this year) | $413,661 |
| NIH project number | 5R01NS136281-03 |
| Project ID | NIH-11314568 on NIH RePORTER |
Who could benefit from this research
✓Could help
People with genetically confirmed LAMA2 (laminin-α2) congenital muscular dystrophy (MDC1A) are the primary candidates for this approach.
✕Less likely to help
People whose muscle disease is caused by other genes or conditions, or who do not have laminin-α2 deficiency, are unlikely to benefit from this laminin-specific therapy.
What this research studies
If you have LAMA2-CMD (also called MDC1A), this project is developing a replacement laminin protein to substitute for the missing laminin-α2 in muscle. Researchers previously gave a form of laminin called laminin-111 to mice lacking laminin-α2 and saw reduced muscle damage, preserved strength, and much longer survival, so the team will refine the protein and dosing for safety and effectiveness. They plan to produce human-compatible laminin, run laboratory and preclinical safety tests, and prepare for possible human studies at clinical sites. Patients would be screened by genetic diagnosis if and when clinical testing begins.
Why it matters
Potential benefit: If successful, this therapy could slow muscle deterioration, improve strength and breathing, reduce feeding or ventilator needs, and extend life for people with LAMA2-CMD.
How similar studies have gone: Preclinical mouse studies of laminin-111 have shown improved muscle pathology and lifespan, but translating this specific protein therapy to humans is still novel.
Where this research is happening
-
University of Nevada Reno
Reno, United States Active
Researchers
- Principal investigator: Burkin, Dean J. — University of Nevada Reno
- Study coordinator: Burkin, Dean J.
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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.