Exon skipping proves effective in mice with a severe DMD-like disease
posted on October 23, 2009 - 10:11am
Mice with a severe disease closely resembling human Duchenne muscular dystrophy (DMD) have responded extremely well to a new "exon skipping" compound that targets the specific dystrophin gene error these mice have.
Researchers noted "a remarkable prevention of the dystrophic pathology and improvement of the muscle function" in these severely affected mice, which lack both the dystrophin and...
Promising minigene only partially normalizes heart muscle
posted on January 21, 2009 - 11:14am
A promising “minidystrophin gene” that restores normal muscle force to skeletal and diaphragm muscles in mice with a disease resembling Duchenne muscular dystrophy (DMD) seems to be only partially effective at restoring strength and function to heart muscles.