Doctors Alter DNA For The First Time Inside Living Human

Move over Doctor Moreau, scientists at the Casey Eye Institute are honing in on your territory, only instead of creating hideous mutants they're trying to cure blindness. According to USA Today, doctors at the Oregon Health & Science University in Portland deployed the gene editing tool CRISPR to treat a genetic form of blindness in a first-of-its-kind attempt to use the promising technology in a living human patient. Researchers did not provide any specific details on the timing of the treatment or the patient profile, but they are hopeful their results will lead to a new generation of treatments for all kinds of genetic diseases.

Way back in 2017, doctors attempted to edit DNA in a live human using an older technology known as "zinc fingers." Interest in the new test results is high because most members of the medical and scientific communities believe CRISPR to be a far more powerful tool, with the ability to precisely cut and replace DNA at a much lower cost than other competing techniques. Although it may take up to a month for researchers at the Casey Eye Institute to report their findings, if their early attempts prove safe they plan to replicate the procedure on a group of 18 children and adults.

The first of many potential genetic treatments

Chief scientific officer at Editas Medicine, Dr. Charles Albright said his Massachusetts-based company is watching the Portland clinic results with cautious optimism. "We literally have the potential to take people who are essentially blind and make them see," he said. "We think it could open up a whole new set of medicines to go in and change your DNA."

This has, of course, always been the promise of CRISPR (short for CRISPR-cas9). Once scientists recognized the gene sequence's potential application as a ubiquitous tool for genetic engineering, research into these potential applications boomed. Thanks to CRISPR, we're living at the dawn of a golden age of genetic engineering.

The patients in the Portland trial all suffer from the same congenital condition: Leber amaurosis. The disease is caused by a single genetic mutation that prevents the body from constructing a necessary protein that converts light from the eye into signals in the brain. Without this protein, Leber amaurosis patients become fully blind within the first few years of life. Doctors hope to use CRISPR to restore this gene to cells in the eye. If successful, they believe that the fix will be permanent.