Preliminary data, released Wednesday, shows results for five people. Two received low doses of the new drug, while the other three received medium amounts. And, while more research is needed, the findings could have far-reaching ramifications for anyone suffering from the condition.
How CRISPR was used to give patients back some of their vision
In previous trials for disorders such as sickle cell disease, gene editing was done in the lab: scientists took patients’ blood, edited the genes in the lab, and injected the “fixed” blood cells back into the bloodstream. . But it was not possible for the treatment of the eye. Thus, the researchers had to devise another method of gene editing. Using CRISPR, a harmless virus that inserts its own genome where the source of ACL disease should reside has been stripped of its genetic material and replaced with the altered sequence that patients need.
But that didn’t solve all the problems. The researchers still had to deliver the virus where it was needed, the retina. It couldn’t be done through the bloodstream, but the cornea is a tough piece of evolutionary craft, so the solution could be injected directly into the cornea.