Laminin protein treatment for LAMA2 (MDC1A) congenital muscular dystrophy
Laminin protein therapy for the treatment of Laminin-alpha2 deficient congenital muscular dystrophy
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 ID | NIH-11314568 on NIH RePORTER |
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.
Who could benefit from this research
Good fit: People with genetically confirmed LAMA2 (laminin-α2) congenital muscular dystrophy (MDC1A) are the primary candidates for this approach.
Not a fit: 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.
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 performed: 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
Reno, United States
- University of Nevada Reno — Reno, United States (Active)
Researchers
- Principal investigator: Burkin, Dean J. — University of Nevada Reno
- Study coordinator: Burkin, Dean J.
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.