Scientists have used a Crispr-Cas9 to edit the DNA and fix genes for the first time inside a living subject. The gene-editing tool Crispr-Cas9 is used to treat diseases that cannot be fixed with conventional means.
One patient got treatment using this procedure at the Casey Eye Institute at Oregon Health & Science University in Portland because of blindness that was caused by genes. The operation was done on a Wednesday with no other details given yet.
A month will be needed to see if the procedure helped restore normal vision. The doctors will only call it safe after a few tries. Once proven safe, it can be used for both adults and children.
Charles Albright, the chief scientific officer at Editas Medicine, which is a Massachusetts-based company developing the treatment said, "We literally have the potential to take people who are essentially blind and make them see. We think it could open up a whole new set of medicines to go in and change your DNA."
Eye surgeon Jason Comander of the Massachusetts Eye and Ear in Boston is getting subjects for the study. He said the treatment is innovative and editing the DNA is easier and effective.
Before doctors tried Crispr, there was an early attempt in 2017 using zinc fingers, but many scientists think Crispr is a better tool to locate and cut DNA at a crucial point. This tool is getting a lot of attention too.
Most of the subjects have Leber congenital amaurosis, that is a mutation that prevents a crucial protein that is needed to convert light so the brain can process the stimuli as sight. Most sufferers cannot see well and become blind soon.
The patient is given general anaesthesia, with three drops that have the gene-editing mechanism that is injected into the retina, and into the light-sensing cells. It takes about 60 minutes to finish the process.
Dr Eric Pierce at Massachusetts Eye and Ear, involved in the first case, said," Once the cell is edited, it's permanent and that cell will persist hopefully for the life of the patient."
It is assumed that fixing one-tenth of the cells will restore vision. During animal testing, half of the cell was fixed by the treatment, Charles Albright remarked.
Little risk is involved in the surgery, and occurrences of infections and bleeding are not so great during treatment.
Making mistakes when doing gene editing is changing the DNA accidentally, but companies are trying to streamline the process and avoid these mistakes.
Dr Jean Bennett, a University of Pennsylvania researcher commented that the gene editing approach is exciting. Technology like it will be make it easier to fix larger genes. She has gotten call from families hoping to get the treatment to avoid getting blind from this genetic illness.
Another expert who knows how to edit a gene and fix part of our DNA, Dr. Kiran Musunuru from the University of Pennsylvania, said the gene-editing tool stays in the eyes only to avoid any irreversible mistakes that can happen.
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