Using gene therapy for the first time managed to prevent the development of blindness

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2017-03-16 18:00:05

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Using gene therapy for the first time managed to prevent the development of blindness

According to the journal Nature Communications, a group of biologists from the U.S. for the first time were able to cure complete blindness in mice. The assurances of the newspaper, it was possible thanks to the use of the editor of the genome of CRISPR/Cas9. With it removed the defective gene responsible for the development of blindness.

Even from a school course of biology, we know that the eyes of some mammals contain in its composition of rods and cones. Cones allow you to distinguish colors and sticks are responsible for the so-called twilight vision, i.e. make it possible to distinguish outlines and objects in low light. But sticks and another function: they support and nourish other cells of the retina, and their destruction leads to the death of the entire retina and vision loss. Most genetic mutations related to loss of vision usually affects the sticks, and the person usually loses the first twilight vision, and then the ability to see the world and daylight color.

How did you find the experts, the growth process of the rods and cones is controlled by the gene Nrl. If this gene is removed by the formation of the embryo, there is the retina, the cones in which is not dead, despite the lack of chopsticks. Based on this, biologists have created a virus with the genomic editor CRISPR/Cas9 that infected and removes the Nrl gene from the DNA sticks. During a series of experiments on three groups of mice with various DNA mutations, deletion of the gene led to a stop of destruction of the retina. In the end, the rodents lost the ability to see in the dark, but did not lose vision in the daytime.

Despite the promise of such technology, the security of the genomic editor CRISPR/Cas9 for use in medical purposes yet to be confirmed, and its use for editing DNA is still not approved and causes some very heated discussions in the scientific community.

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