For example, researchers would like to know how the drug
affects dystrophin production in muscles throughout the limbs and whether some muscles may get a bigger boost than others.
Cardiologist Kirk Knowlton of the University of San Diego and his colleagues wanted to find out whether the Coxsackie B virus also
affected dystrophin.
Not exact matches
The result was the largest deletion ever observed in the
dystrophin gene using CRISPR / Cas9, and the study was the first to create corrected human iPS cells that could directly restore functional muscle tissue
affected by Duchenne.
DMD is a rare disease
affecting primarily boys and is caused by defects in the gene that makes the
dystrophin protein.
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.
If any one exon gets a debilitating mutation, the chain does not get built.Without
dystrophin providing support, muscle tends to shred and slowly deteriorate.Duchenne
affects one in 5,000 newborn males.