A new study shows that muscular dystrophy in dogs can be treated by gene editing. CRISPR/CAS9, which is a gene editing tool, has allowed scientists to rectify Duchenne muscular dystrophy (DMD) in dogs. The study was published in the Science Journal, titled, “Gene editing restores dystrophin expression in a canine model of Duchenne muscular dystrophy.” DMD is the most common form of muscular dystrophy, a term describing various conditions that progressively destroy muscles throughout the body. This deterioration of the muscles is primarily caused by the inability to use dystrophin, a protein that acts as a glue to stabilize muscle fiber.
These conditions are generally considered a genetic defect, with the disease primarily affecting males. This is due to the fact that the disease affects one X chromosome which affects men more as women have two copies of X chromosome. A woman with a single copy of the defective chromosome may show mild symptoms, but this occurs on rare occasions. However, women usually act as carriers by passing on the disease to their sons. The disease also affects animals, including dogs.
The study states that “this is an essential step toward clinical translation of gene editing as a therapeutic strategy for DMD.” However, the use of gene editing for curing DMD in humans is still a dream. Dr. Eric Olson the lead author of the study states that it is “too early to test this technology in humans. We need to determine safety and durability before trying it in patients,” adding that this will take “several years.” Furthermore, even if it is safe to test on humans, there is no guarantee that this method will work just because it has worked on dogs.