CRISPR , the gene editing technology, was used for the first time in the world for therapeutic purposes. According to the statements of the two companies carrying out the treatment, the treatment performed at the Casey Eye Institute of Oregon Health and Science University in Portland, USA , aims to treat congenital blindness with CRISPR .
According to the statement of Cynthia Collins , CEO of Editas Medicine, one of the companies working in the field of gene replacement, this treatment will be very beneficial both for medicine and science and for people with eye disorders.
Treatment with CRISPR was performed on a patient with leber congenital amorosis , a congenital genetic disease . This disease is caused by a faulty gene, and the disease affecting about one in 40 thousand children today has no cure.
The genes that are wrong with CRISPR can be corrected by injecting the experimental drug named AGN-151587 into the eye of the patient . Thanks to CRISPR, changes can be made in DNA by removing the mutation that occurs in the genes . Moreover, the effect of this change with CRISPR is permanent.
In the experiment using CRISPR , different doses will be applied in 18 patients in total, and thus it will be understood how much dose is needed to correct blindness. Although the dose used in the first patient treated is insufficient, detailed information about the patient is unknown. This treatment, which has just begun to be tried, is thought to be a hope for patients.
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