Could Gene Therapy Work for Muscular Dystrophy?
One of the most common and severe forms of genetic disorder is the DMD or Duchenne Muscular Dystrophy. For decades, the scientists have been trying to find a solution that would restore the muscle strength and stabilize other symptoms associated with this serious illness.
Now, the latest findings in gene therapy applied in dogs are giving us hope that we can finally do something about the DMD. There’s an optimistic expectation that one day we can use gene therapy on children to treat this condition. The Duchenne Muscular Dystrophy affects approximately one in 50,000 children, mostly boys, very rarely girls. The first signs of the disease become apparent between the age of two and five.
The DMD influences the dystrophin, which is a protein responsible for our muscle strength and integrity. As a result of this disruption, our muscles become weak. By the time a boy affected by the DMD reaches the age of 12, he's unable to walk on his own.
Gene Therapy vs. DMD
In order to deal with a genetic disorder, the researchers put a healthy alternative of the affected gene into a vector (usually a virus, but a harmless one), which delivers the material into the affected cells. Unfortunately, when it comes to the DMD, there’s a huge problem. The target gene has more than two million base pairs, which makes it to big to be delivered by a vector (virus).
One of the ways to solve this problem is to create a new shortened version of the target gene – dystrophin. That’s something the researchers are already working on.
Currently, they have managed to come up with the shortened version that has only 4,000 base pairs, which is a huge success compared with the initial number of two million base pairs. The goal is to make sure that the functional proteins are produced again.
For the purposes of the gene therapy research, a dozen of golden retrievers were chosen. These dogs had a natural DMD predisposition. The microdystrophin was injected into the dogs intravenously.
Then, the scientists have observed the changes for the next two years. Pretty soon, it became obvious that the production of dystrophin was restored to the normal levels. In addition, the muscle functions were significantly restored too. Other symptoms were stabilized, as well. The most important thing is that there were no side effects associated with this gene therapy approach.
The Man’s Best Friends Have Shown Us the Way
The importance of this research is that we have never before conducted gene therapy on large animals. The optimism comes from the fact that there similarities in size and symptoms between children and dogs. The scientists believe that we aren’t too far from the moment when we will be able to use gene therapy on humans full-scale.
The efficiency and safe character of gene therapy with no known side effects are just the things we need to deal with the DMD. So, what's stopping us from trying the same thing on patients? In order to be absolutely sure about the positive outcome, we need more successful trials.
For what is worth, this is the first time that we were able to treat the large-sized animals in this way with the help of gene therapy. The great thing about it is that we can treat all DMD patients, regardless of the genetic mutation variations.
It is quite obvious that in gene therapy the size matters. The test subjects have to be approximately the same size as the affected children. Otherwise, we can never be sure that the gene therapy on children will work for sure.
The videos of the dogs that received treatment and were able to jump over the plastic gates were a huge encouragement for both scientists and parents, who’re looking forward to seeing a solution, which can help their children.
How Long Will We Have To Wait?
The biotechnology companies and the pharmaceutical industry itself are impatient to begin with the real-life applications. There are even extremely optimistic plans to restore the entire form of the dystrophin and increase the chances of quick and complete muscle recovery.
For the scientists involved in trials on large animals, there's no room for doubt. We're literally one step away from the trials on boys struggling with the DMD. The trouble is that this step can be easily stretched to a couple of years. We are impatient and full of hope, but still, we're hesitating.
It's in our human nature to question the breakthrough discoveries that have the potential to revolutionize the way we live and treat serious conditions. It is in our genes. Can gene therapy treat this gene of the doubt? It remains to be seen. Until then, both our fingers and paws are crossed.
Read Next Article Is Gene Therapy Safe?