Experiments have shown treatment with sildenafil significantly improved heart function in mice
missing the dystrophin protein.
«We took patient - derived cells that had the most common mutation responsible for Duchenne muscular dystrophy and we corrected them in vitro to restore production of
the missing dystrophin protein in the cells.
Not exact matches
The exact mutation varies from patient to patient but in 65 percent of cases, the
dystrophin gene is
missing large sections of DNA called exons, which carry the instructions for protein production.
Using the natural human development process as a guide, the researchers developed ways to mature muscle cells in the laboratory to create muscle fibers that restore
dystrophin, the protein that is
missing in the muscles of boys with Duchenne.