Duchenne muscular dystrophy or DMD is a serious genetic disorder that affects hundreds of kids each year. This disorder results in ailments like atrophy and muscle fatigue. What causes Duchenne muscular dystrophy so harmful for kids? Kids, suffering from DMD cannot create dystrophin, a protein that helps to keep the muscles away from breaking.
The main reason is, these folks have gone through a mutation within their gene in the X chromosome, which is responsible for providing the blueprint for dystrophin. The genetic mutation may result in a more serious situation where most regions of the dystrophin get influenced and a result the body stops producing the protein completely. For many people with DMD, there are a lot of errors in the gene the body cancels out the affected protein that the moment it is created.
However, what is more interesting to see is, the investigators have focused on the genes which are entirely flawed and that disrupt in reading blueprint to make dystrophin. So as to deal with the criticalness of the condition, researches have conducted a research on a mice model. Multiple groups of researchers have exploited the technique of employing gene editing enzyme complex CRISPR so as to alter the genes of mice with DMD.
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The technique allows cutting the mistakes in the genetic code, so that their consequences can just be avoided. After that the body begins to read the gene and produce a briefer version of the protein which manages to protect the muscles from breaking down so easily, much like the procedure to take care of patients with Becker muscular dystrophy. The procedure involves loading the CRISPR complicated to a virus and then injecting it to the mice fetus with DMD.
The researchers have discovered that the mice started to make the shortened protein when the virus is injected into them. This helped the mice to restart the power of the muscles, which gradually led to a concrete treatment for their disorder. So, the research has been quite successful on the mice model.
The findings have generated a thin window of hope for those, suffering with Duchenne muscular dystrophy mutation also. But the procedure was simpler for mice, as well as the investigators know that it is not going to be the identical simple game for humans. Moreover, no matter how foolproof the procedure is proved for the mice, it may not provide the exact results on humans. The scientists have established the CRISPR can cut off additions to the gene but it will still take a while to reveal if it can address different kinds of mutations in any respect.
Researchers are also unsure about the responses of CRISPR complicated in humans. Chances are that they may get rejected by the human immune system entirely. If it doesn’t end up being a 100% cure to the disease, it means that the procedure won’t be able to take care of all patients with Duchenne muscular dystrophy mutation. However, having said all these, it’s also true that this study is the first ever experiment to have shown success, even though on a mice model. And researchers are optimistic that with more clinical trials, they may attain the desired results shortly.