CRISPR is coming for your body. While that may sound like the tagline of a horror film, it’s potentially the future of medicine, as the gene-editing technique is about to be used for the first time inside the bodies of presumably willing patients.
The first trial could be an impressive feat of modern medicine: Scientists hope to use CRISPR’s gene-editing magic to restore sight to people with an inherited form of blindness called Leber congenital amaurosis. It’s the most common cause of inherited childhood blindness, occurring in about 2 to 3 of every 100,000 births, according to the AP.
People with the disease lack just one gene, the gene that converts light into signals to the brain and lets the eyes do their job. The hope is that adding that gene through a one-time CRISPR treatment will cure them, permanently editing their DNA and letting them see. This is undoubtedly a more noble use of CRISPR than making spicy tomatoes.
It’s a good trial for the cutting-edge—and controversial—technology, because there’s already some solid proof that the disease is treatable on a genetic level. As the AP notes, thanks to a gene therapy called Luxturna, which is already on the market, scientists know that injecting a replacement gene into cells in the retina can cure the ailment. CRISPR would work by similarly delivering new genetic material to the eye, giving researchers the perfect testing ground for using the new medical procedure in humans, before turning to more complicated diseases like cancer.
Two companies, Editas Medicine and Allergan, will test the technique in up to 18 people at hospitals around the United States, starting this fall. The new study will test children at least 3 years old and adults with a range of vision. According to the AP, only one other company has tried gene editing inside the body before: Sangamo Therapeutics uses the technique to treat metabolic diseases with a tool alluringly called zinc fingers.
CRISPR watchers may be wondering how this study can be considered the first trial in humans, because a Chinese scientist controversially used the technology to alter the DNA of two embryos, who were born last year and given the names Lulu and Nana. For this new study, scientists aim to alter the DNA in humans who have already been born. The changes theoretically will not be inherited by any offspring. Also, this one is not being done in secret, and so hopefully no one will lose their job.