Editor of genome CRISPR managed to cure muscular dystrophy

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2018-08-31 22:15:07

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Editor of genome CRISPR managed to cure muscular dystrophy

Editor of CRISPR genome has been repeatedly successfully used to treat a variety of anomalies. And now the editors of the journal Science Alert reports that CRISPR again showed its effectiveness. This time in the treatment of Duchenne muscular dystrophy.

Duchenne Muscular dystrophy (it is called muscular dystrophy Becker) is a genetic disease associated with mutations in the gene DMD, which encodes the protein dystrophin. Disease linked with X chromosome and is characterized by proximal muscle weakness caused by degeneration of muscle fibers. Such patients are at an early age lose the ability to walk, and to 30 years the majority of developing cardiopulmonary failure, which often is the cause of death. All complicated by the fact that the DMD gene consists of many "parts" (exons) which can be damaged in the process of formation, and that will cause a defect in the gene.

Scientists from the University of Texas together with their colleagues from the Royal veterinary College London used a fairly interesting approach, called the omission of exons (exon skipping). It lies in the fact that in 13% of patients reading gene can be restored by removal of exon 51. The studies involved dogs of the Beagle breed who have the same disease and for his development is in the same gene, the damage of which leads to the same consequences.

Schema recovery of dystrophin gene with exon skipping or restore its consistency

With Only CRISPR system was conducted 2 experiments: in the first two puppies from the gene affected in DMD calf muscle introduced viral particles with CRISPR and after 6 weeks had a test measurement. It turned out that in the muscle tissue began production of dystrophin in total, 60% of normal. In the second experiment, two other puppies entered in the blood of the same particles that were injected into the muscle. After 8 weeks, the number of dystrophin reached concentrations up to 50% of normal have 1 puppy and up to 90% of normal at the second puppy.

However, even despite such impressive results, according to scientists, the therapy using CRISPR requires an additional and extremely long trials, because at the moment it is not clear how long the concentration of protein in the muscles is maintained at the correct level and how it affects the body as a whole.

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