Overview
This study investigates the role of High Mobility Group Box Protein 1 (HMGB1) as a potential biomarker for Duchenne Muscular Dystrophy (DMD). The researchers utilized RNA sequencing in mouse models and human iPSC-derived skeletal muscle cells to assess the expression of HMGB1 under DMD conditions.
Key Findings
- HMGB1 Levels in DMD: The study found that HMGB1 is elevated in DMD conditions.
- Impact of Gene Therapy: Microdystrophin gene therapy successfully reduced HMGB1 levels in treated models.
- Comparison with VCAM1: The study also examined vascular cell adhesion molecule 1 (VCAM1), but found it to be less reliable as a biomarker for DMD.
Conclusion
HMGB1 appears to be a promising biomarker for DMD, particularly in monitoring disease progression and evaluating the effectiveness of gene therapies like microdystrophin. The study highlights the need for further research to validate HMGB1 and explore its potential clinical applications.
| Published | 8/19/2024 |
| Address | https://doi.org/10.1242/bio.060542 |
| Authors | Rebecca A. Slick1,2,3, Jessica Sutton1,4, Margaret Haberman1,4, Benjamin S. O’Brien4 , Jennifer A. Tinklenberg1,2,3, Aashay Mardikar1 , Mariah J. Prom1,4, Margaret Beatka1,4, Melanie Gartz1,5, Mark Vanden Avond2 , Emily Siebers1 , David L. Mack6,7,8, J. Patrick Gonzalez9 , Allison D. Ebert5 , Kanneboyina Nagaraju10,11, Michael W. Lawlor1,2,4,* |
