Up to 18 people ages 3 and up around the United States will participate in the first study to test a gene-editing technique inside the body. The technique known as CRISPR will be used to try to cure an inherited form of blindness. Massachusetts Eye and Ear in Boston and two companies, Editas Medicine and Allergan, will be the ones testing this procedure. Before this, there has only been one other company, Sangamo Therapeutics, to attempt gene editing inside the body. In that case, it was to treat metabolic diseases using a tool called zinc fingers.
People with the vision disease, Leber congenital amaurosis (LCA), have normal eyes but lack a gene that converts light into signals to the brain that enables sight. This heritable disorder is the number one cause of inherited blindness in children, occurring in about 2 to 3 of every 100,000 births. People often only see bright light and blurry shapes if anything and then eventually can lose all sight. An individual is born with this form of blindness when both parents are carriers of the flawed gene. The combination of getting two copies is what causes it.
The experimental treatment aims to supply the patients with a healthy version of the gene they lack. They will do so by using a tool that cuts or “edits” DNA in a specific spot. It will be administered through a subretinal injection. The process is intended as a one-time treatment since it permanently alters the person’s native DNA. Although, this change will not be passed on to future generations, setting the treatment apart from the highly controversial series of human embryo trials by Chinese scientist He Jiankui, who used a similar technique to edit the germline of future human generations.
Many scientists are captivated by the potential of CRISPR. It is a very simple way to do gene editing, although it’s so new that its risks are not fully known. Nevertheless, researchers believe it shows great promise to cure or treat many diseases caused by genetic flaws, especially those that have no good treatments now. Hopefully, this experimental trial works out well and gives the participants the gift of sight, thus proving the benefits of CRISPR treatments.
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