Exon skipping proves effective in mice with a severe DMD-like disease
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...
The underlying cause of Duchenne muscular dystrophy (DMD) can be any of a large number of mutations in the gene for the dystrophin protein. One strategy now being tested for overcoming this problem is gene therapy, the insertion of new dystrophin genes. Another is the transplantation of cells that give rise to muscle.
Stephen Wilton, an MDA grantee, and colleagues at the University of Western Australia in Perth, with scientists at AVI BioPharma in Corvallis, Ore., report significant progress in two sets of experiments that make use of the technique known as exon skipping.