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Patients in the U.S. are about to be the first enrolled in a gene-editing technique, known as CRISPR, used inside the body to help treat them of a specific inherited form of blindness.
People with this disease have regular eyes, but are missing a gene that allows light to be converted into signals sent to the brain, and which allows them to see.
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This experimental study will involve both children and adults in the treatment, in order to restore vision in their eyes.
Gene in a specific spot
CRISPR will edit the gene these patients lack in order to see, by cutting and editing DNA in a specific place. It's meant as a one-time operation, which alters the person's DNA forever.
Editas Medicine has made it. The gene-editing biotech firm is moving their treatment for a rare form of blindness (LCA10) into human testing. The FDA signed off on the phase 1/2 in November, and the partners have started enrolling patients. #moderntrials#clinicaltrialspic.twitter.com/YPZZ1edopa— Modern Trials (@ModernTrials) July 27, 2019
CRISPR is of particular interest to scientists around the world, as it is simple and can edit our genes. However, side effects and reactions still remain unclear at this point.
The two companieswho will be carrying out this specific test in the U.S. are Editas Medicine and Allergan. Eighteen people will be tested around the States starting in the fall.
By editing these patients' genes, it will hopefully stop the disease from moving down generations.
The eye disease
The disease is called Leber congenital amaurosis, and those who will be taking part in the blindness study have one form of it. It is the most common cause for blindness inherited in children, occurring in around two to three out of every 100,000 births.
The way people are affected by the disease is that they typically can only see a bright light and blurry shapes, eventually losing sight altogether.
As it is inherited, parents can carry the genes, sometimes in dormancy for generations, before passing it on to their offspring.
This new study will test children from three years and up, as well as adults, all with varying vision.