General, Research, Technology

Scientists Cure Blindness Using Gene Editing Technology

Famous CRISPR Gene Editing Technologywas first used inside the human body. Scientists from the Oregon University of Health and Science (OHSU) presented to the public the results of a new experiment, during which a progressive method of treating hereditary blindness was invented, reports portal Despite the fact that genetic engineering as a science appeared in the middle of the twentieth century, scientists for a long time did not dare to apply its discoveries to humans, fearing the emergence of a number of ethical problems that accompany this branch of knowledge. Be that as it may, a recent human operation can give green light to the active use of CRISPR in the treatment of a number of diseases. Is gene editing beginning to gradually enter our daily lives?

Gene editing technology was first applied in the treatment of blindness.

Gene editing technology has helped in the treatment of vision

Amabrosis of Leber - a hereditary disease,due to which patients are born blind or lose their sight during the first years of life. This rare disease is caused by the occurrence of a special genetic mutation that negatively affects the retina, ultimately depriving the patient of the opportunity to see. In order to try to correct a defective gene that causes the appearance and further development of the disease, scientists first applied gene editing technology within the walls of the University of Oregon by injecting a charged CRISPR drug directly into the photoreceptor cells behind the affected retina.

See also: CRISPR genome editor for the first time helped to destroy the sleeping forms of the herpes virus

Since its development in 2012,Gene editing technology has proven to be a promising way to treat a range of genetic diseases. The new tool allows scientists to literally cut out damaged sections of DNA that become the cause of the development of the disease, inserting something more useful into an empty space.

A new study at OHSU aimed at treating Leber's congenital amaurosis syndrome

During the preparation for the experiment wasLeber amaurosis was found to be caused by a malfunction in the CEP290 gene, which is responsible for retinal development. Although CRISPR technology has been tested in humans before, surgery on organs of vision affected by hereditary syndrome was the first human-made editing. So, if earlier doctors had to remove cells from the patient, editing them in the laboratory and then returning them to their place, the new clinical trial marks the first time that CRISPR has been edited directly inside the human body.

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Inspired by the success of the operation, MarkPennesi, a leading OHSU scientist, noted that already in the relatively near future, CRISPR technology will be able to open up almost unlimited prospects in the treatment of genetic diseases. In addition to preventing hereditary blindness, gene editing directly inside humans can also save us from a much wider range of diseases than previously thought.

As it often happens, the application of a newThere is one significant “but” of the treatment method. In this case, experts fear that artificially edited genes may affect children, which patients will probably want to have in the future. If during the operation the doctor makes even a small mistake, the consequences of incorrect editing can change the natural gene pool of a person for generations.

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Be that as it may, application of CRISPR technologySo far, only one patient has been carried out, so it is too early to talk about the final results of gene therapy. Scientists noted that in the near future 18 more volunteers are awaiting treatment, ready to tell in the future about the effectiveness of using a unique technique.