Researchers have used CRISPR to treat Duchenne muscular dystrophy in four dogs, according to a study published yesterday (August 30) in Science. By editing cells in one-month-old beagles serving as models of the disease, the team boosted the expression of the gene coding for dystrophin—a protein whose dysfunction underlies Duchenne muscular dystrophy (DMD)—to up to 92 percent of normal levels in some tissues.
“This is a very exciting paper as it shows that gene editing can be reasonably [effective] in a large animal model of DMD,” Kay Davies, director of the MRC Functional Genomics Unit at the University of Oxford and who was not involved in this work, tells GEN.
DMD is a rare, X-linked condition. The one currently approved gene therapy, Sarepta Therapeutics’s eteplirsen (Exondys 51), can only restore dystrophin to around 1 percent of normal levels—and only in some patients.
Previous work by the same authors of the ...
