Substituting the Next-Best Protein

Posted on Friday, April 24, 2020

When an actor is unable to perform in the theatre, an understudy—ideally one with some practice in the role—can take her place on stage. A study from Dr. Bernard Jasmin’s laboratory and published today in Nature Communications shows that the same is true of proteins. Its results point the way toward novel therapies for Duchenne muscular dystrophy.

Children born with Duchenne muscular dystrophy (DMD) have a mutation in the X-chromosome gene that would normally code for dystrophin, a protein that provides structural integrity to skeletal muscles. The loss of this protein causes severe symptoms, including deteriorating muscle strength beginning around the age of four. The average life expectancy of a child with this condition currently stands at 26 years.

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