It's important to remember that we're not going after the primary cause of the disease,
dystrophin deficiency.»
Not exact matches
This approach has proved successful in disrupting multiple genes simultaneously in neurons and in editing the Dmdmdx mutation in muscle, thereby restoring
dystrophin protein expression and partially alleviating the dystrophic muscles» functional
deficiencies.
Duchenne muscular dystrophy, which strikes in 1 out of 5,000 newborn boys, results from a genetic
deficiency in
dystrophin, a protein involved in repairing muscle fibers affected by daily movement and activities such as exercise.