Becker muscular dystrophy (BMD) is a genetic disease that affects muscles, caused by mutations in the dystrophin gene. It's not as severe as Duchenne muscular dystrophy (DMD), but there aren't many treatments available for BMD. This is partly because there's no good mouse model for studying the disease and testing potential treatments before trying them on people.
To address this, researchers created a mouse model called bmx, which mimics BMD by deleting specific parts of the dystrophin gene. They then studied the muscles and hearts of these mice using various techniques.
The bmx mice showed muscle weakness and heart problems similar to BMD patients, but not as severe as in mice with complete dystrophin deficiency. Their muscles had signs of damage and regeneration, as well as some inflammation and scarring, similar to what's seen in BMD patients.
This mouse model can help researchers understand BMD better, find potential treatments, and test them before moving to human trials. It's a valuable tool for studying the disease and developing therapies for BMD patients.
Published | 11/1/2023 |
Address | DOI: 10.1002/jcsm.13171 |
Authors | Christopher R. Heier1,2 , Nikki M. McCormack1, Christopher B. Tully1, James S. Novak1,2, Breanne L. Newell-Stamper3, Alan J. Russell3 & Alyson A. Fiorillo1,2* |