Sunday, September 23, 2018
CRISPR Gene Editing effective in treating muscular dystrophy in dogs
CRISPR gene editing is a very new method and has limitless potential in the medical field to treat diseases related to genetics. This technique uses a man-made molecule (CRISPR) with complementary base pairs to a DNA sequence of interest. This molecule locates the target DNA sequence and can either delete or edit the DNA sequence. The implications of this method are endless, and scientists want to begin using it to treat genetically based diseases such as sickle cell anemia (which is caused by a single base mutation) and muscular dystrophy.
Muscular dystrophy is caused by a mutation in the gene dystrophin, which is responsible for normal muscle function. In individuals with muscular dystrophy, over time their muscles begin to rapidly degrade and usually end up confined in a wheelchair, because of loss of leg muscle. These individuals also have shorter life spans because they eventually end up on a respirator because even the muscles of the lungs and diaphragm fail. The symptoms first start to appear by childhood and the individual may end up in a wheelchair as early as their teen years.
Because this disease is caused by genetic mutation, scientists have thought about using CRISPR gene editing techniques to possibly treat this disease. Previous studies have successfully treated the disease in rodents, but this article discusses a very recent finding in which they treated dogs with CRISPR gene editing for their muscular dystrophy. This was the first time they have successfully used CRISPR gene editing on a larger mammal species. They used the gene editing method to splice out a mutated part of the gene and analyzed the lasting effects on the dog's health. They tried injecting CRISPR directly into the muscle and also into the bloodstream. They found that after direct injection in the muscles, the dogs began synthesizing normal healthy dystrophin protein in the range of 3-90% of the normal levels for those specific muscles.
Although this is the very early stages of this research, only a few dogs actually received treatment in this study, it shows very promising results. This could be very beneficial because once the symptoms start in humans, we could apply this sort of treatment before the symptoms become much worse and ultimately confine the person's life and stop their heart and lungs from functioning. Another important note to focus on is that the treatment only saw increased dystrophin levels in the muscles that were directly treated with CRISPR. Another method would have to be investigated to discover a more efficient way to modify the genes in the more centralized organs such as the lungs and heart. It is also important to investigate the lifelong health effects that this treatment may have on dogs and other model organisms to determine if the treatment is safe for human use. I look forward to reading more about the progress of this research because it is definitely heading in the right direction and I hope it proves safe for human use because it could truly be a life-changing treatment and forever change the way we view medical treatments.