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Human clinical trial of gene editing will be launched in the United States
Release Time:12.07.2018 Posted by:group headquarter

A gene editing company has announced that it will launch a clinical trial of CRISPR gene editing technology to treat some hereditary eye disease. The application has been accepted by US regulatory authorities.




It is reported that in this clinical trial, the target of gene editing is the photoreceptor cells in the eyes of patients with congenital amaurosis, which is a kind of somatic cell rather than a germ cell. The genetic information of somatic cells will not be passed on to the next generation, so it does not involve ethical issues.




The treatment, called edit-101, was developed by edditas and elgene. The treatment "is expected to be the first in the world to use CRISPR technology in humans," edditas said in a statement.




The statement said the U.S. Food and drug administration has accepted the company's clinical trial application for the treatment, allowing it to use CRISPR technology in the treatment of Lieber congenital amaurosis type 10 patients.




Lieber congenital amaurosis is a hereditary retinal degenerative disease caused by multiple gene mutations. It is the most common cause of congenital blindness in children. It affects 2 to 3 children per 100000 children worldwide. Among them, type 10 is the most common type, accounting for 20% to 30% of the total number of patients with this genetic disease. At present, there is no effective treatment for Lieber congenital amaurosis.




According to the plan, the clinical trial will recruit 10 to 20 patients to test the safety, tolerance and effectiveness of edit-101 therapy.