src="https://webmd-a.akamaihd.net/story-telling/st-73fbcd99-a2ad-eb11-8d57-aa6cb854a633/0059284/storage/app/media/crispr-baby-puzzle-loop.mp4" type="video/mp4">In July 2019, Victoria Gray woke up in a sunlit, pale green hospital room at Sarah Cannon Research Institute in Nashville. A frozen bag of her genetically edited blood cells thawed by her bed. Gray, a 34-year-old mother of four from Forest, MS, was about to become a medical trailblazer.
Gray inherited her disease from her parents. It's passed down through recessive sickle cell genes from the mother and the father. Any child born to two people who carry the malfunctioning gene but who don't have SCD has a 25% chance of having the condition. If both parents have SCD, all of their children will, too. The disease strikes mainly people who trace their ancestry to Africa, where the sickle cell gene is thought to have protected against severe malaria.
The splicing technique used to override the genomic codes responsible for Gray's illness is CRISPR-Cas9. It's a simpler, cheaper, and more precise version of the genetic editing technologies that surfaced in the 1990s but never lived up to the hype. About a decade ago, Jennifer A. Doudna, PhD, was studying bacteria in petri dishes when she deciphered how they fought off viruses. Specifically, the biochemist found that these and other microorganisms defend against infections by memorizing the DNA sequences of previous invaders so that when they return, the immune system is precisely primed to vanquish them.
"It allows you to alter the blueprint of life for any organism you want," says Eric Olson, PhD, a molecular biologist at the University of Texas who is developing CRISPR-based treatments for muscular dystrophy."You can't really overstate the potential long-term impact of this technology on life as we know it."
But it wasn't long before another scientist, this time in Russia, began telling reporters he wanted to use a similar embryo-editing plan to enable couples who carry a gene for congenital deafness to bear children with normal hearing. Alternatively, in 2020, doctors in Oregon dripped liquid embedded with the CRISPR tool into the retina of an adult who was born with a rare type of hereditary blindness. The drops were designed to repair a gene called CEP290 to restore eyesight. Children and adults with blindness around the world are awaiting preliminary results, due out later this year.
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