Gene-editing is considered the pinnacle of modern medicine.
Scientists theorize that, with the right information on the exact genetic makeup of the body, we could potentially eliminate every disease known to man.
And curing blindness might be up first.
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Two companies now have the first-ever trials lined up to treat Leber congenital amaurosis. It's the most common cause of inherited childhood blindness, according to the AP occuring in about 2 to 3 of every 100,000 births.
The condition is caused when people are born without a particular gene, one that converts light into signals the brain can interpret. Think of it like trying to use a digital camera that's had its software wiped. But using the gene-editing CRISPR technology, scientists can technically add that gene to a person's makeup, permanently curing this type of blindness.
It's a noble ambition for a first trial, one that will be carried out on volunteer human patients. And that's only because previous studies have shown concrete proof that the disease can be treated on a genetic level. Previous gene therapy called Luxturna, which is already available in the market, has shown it can cure the ailment by injecting a replacement gene into cells in the retina. The CRISPR treatment would work the same way.
The idea to test the new method then is simple. If it works, it gives scientists the reassurance they need to use CRISPR to tackle more complex diseases like cancer.
Two companies, Editas Medicine and Allergan, will test the new technique in up to 18 people around the United States starting later this year. Adults and children at least three years old will be admissible, and of course it's all completely voluntary.?
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That's because there are still risks. Take for instance the recent case of a Chinese researcher that used CRISPR to try and immunize two baby girls to HIV. He did succeed in his ambition. However, because genes often work to multiple purposes, he may have also shortened their lifespan without meaning too.
That's why this sort of trial is better. It's starting at a smaller scale, and it's happening with full disclosure in a way that can be monitored.