Duchenne’s myodystrophy is one of the most common and devastating genetic diseases of muscle. It significantly reduces the quality of life of patients and duration. More recently, however, scientists from the technical University of Munich (TUM) used the gene editing tool CRISPR to cure this disease Guinea pigs. This means that a step closer began testing a similar technology on humans.
Explain that a protein called dystrophin is needed for formation of healthy muscle tissue. But in people with Duchenne’s myodystrophy have genetic mutations that prevent the production of dystrophin.
typically, muscular weakness begins to manifest by the age of five to 12 years the patients stop coming. In addition, such people rarely live up to 30 years.
As noted above, Duchenne’s myodystrophy, is a genetic disease. For this reason, specialists try to cope with it by means of editing technology CRISPR genes. “News.Science” (nauka.vesti.ru) told me about this tool that has been used for the treatment of cancer, HIV and some forms of blindness.
with the recent work of German specialists conducted an experiment on pigs. With CRISPR, they “corrected” the defective gene of dystrophin. As a result, the body of pigs has once again started to produce the necessary proteins. The last was shorter than usual, but was stable and was functioning well.
But most importantly, this procedure has led to improved muscle function and lifespan of the animals. Moreover, the intervention also reduced the likelihood of pigs arrhythmia.
the Disease more often affects boys and very rarely girls.Photo Global Look Press.
there is more. Such treatment allows to avoid one of the main problems that often occur when using CRISPR, — kratkofilnasty effect. The fact that the cells in our body are constantly updated, and therefore, those that are edited, sooner or later die, making any treatment ineffective. But the cells of the myocardium – middle muscular layer of the cell — big “life”. Therefore, the effect of their changes will be preserved longer.
By the way, in earlier experiments, the researchers used CRISPR to cure Duchenne’s myodystrophy mice and dogs. But the body of the pig in many respects much more similar to the human body, so a recent study closer to the clinical testing of the technology.
Scientific article on the results of a study published in the scientific journal Nature Medicine.
Earlier, the “News.Science” (nauka.vesti.ru) wrote that people suffering from Duchenne muscular dystrophy, the new exoskeleton will help to learn how to manage their hands and also about why some dogs have no signs of occurrence of muscular dystrophy, despite them inheriting the faulty gene.