After using the CRISPR gene-editing tool to restore a muscle protein that causes muscular dystrophy in dogs, researchers are hoping the approach can be used on humans who suffer from the genetic disorder, according to a study.
In a recent study published in Science, a research team looked deeper into dystrophin, which is described as a protein that maintains muscle integrity and function—and when mutated, it causes Duchenne muscular dystrophy (DMD), a genetic disorder that causes progressive muscle degeneration and weakness.
According to the study, researchers used adeno-associated viruses to “deliver CRISPR gene-editing components to four dogs and examined dystrophin protein expression six weeks after intramuscular delivery or eight weeks after systemic delivery.”
“After systemic delivery in skeletal muscle, dystrophin was restored to levels ranging from 3 to 90 percent of normal, depending on muscle type. In cardiac muscle, dystrophin levels in the dog receiving the highest dose reached 92 percent of normal,” the study said. “The treated dogs also showed improved muscle histology. These large animal data support the concept that, with further development, gene editing approaches may prove clinically useful for the treatment of DMD.”
Researchers are also hopeful this achievement may have positive implications for humans who also suffer from muscular dystrophy.
“The feat—achieved for the first time in a large animal—raises hopes that such genetic surgery could one day prevent or treat this crippling and deadly disease in people. An estimated 300,000 boys around the world are currently affected by DMD,” a report on the study said.
Danielle covers Clinical Innovation & Technology as a senior news writer for TriMed Media. Previously, she worked as a news reporter in northeast Missouri and earned a journalism degree from the University of Illinois at Urbana-Champaign. She's also a huge fan of the Chicago Cubs, Bears and Bulls.