Not a week goes by without a breakthrough involving CRISPR. From treating blood disorders to genetically editing mutated cancer cells, CRISPR research has yielded encouraging results in recent years. Scientists have now managed to give some of their vision back to patients suffering from a rare genetic disease called Leber congenital amaurosis, or LCA, which starts to impair one’s sight at birth, and progressively gets worse, eventually making them legally blind.
The trial was done in collaboration with the Casey Eye Institute at the Oregon Health & Science University and the gene-editing biotech startup Editas Medicine. After it was seen that preliminary trials on mice and non-human primates were both safe and encouraging, a clinical trial on humans was organized. . Two were given low doses of the new medication, while the other three received mid-range amounts.
But that didn’t solve all the problems. The researchers still had to deliver the virus to where it was needed, the retina. That couldn’t be done through the bloodstream, but the cornea is one resilient piece of evolutionary craftsmanship, so the solution could be directly injected into one’s cornea.Researchers were able to inject a small amount of the solution containing the virus between the cornea and the iris.
Carlene Knight , from Portland, is happy about the improvements. Her vision has become clearer and brighter, and she can now look for objects instead of aimlessly reaching around., Knight said,"I've always loved colors. Since I was a kid, it's one of those things I could enjoy with just a small amount of vision. But now I realize how much brighter they were as a kid because I can see them a lot more brilliantly now. It's just amazing.
While the treatment is far from curing the patients in the trial, some of the changes experienced by the patientssince they are profound enough to have an impact on their daily lives. More patients must be treated and observed for a longer period of time to confirm the treatment's safety, and the researchers have been given permission to proceed to the next group of patients.