The FDA approved two gene therapies for anyone 12 and older suffering from the most severe form of sickle cell disease, a brutal blood disorder that has long been neglected by medical research.

"I'm elated, excited, in awe," Jennifer Doudna of the University of California, Berkeley, who helped discover the gene-editing technique called CRISPR that is used in one of the sickle cell treatments, told NPR in an interview.

Gene-editing, which allows scientists to manipulate the basic building blocks of life more easily than ever before, is being studied as a treatment for illnesses ranging from rare genetic disorders like muscular dystrophy to common ailments like cancer, heart disease, diabetes, AIDS and Alzheimer's.

"I'm really excited," Dr. Lewis Hsu, a pediatric hematologist at the University of Illinois at Chicago who serves as the chief medical officer at the Sickle Cell Association of America, told NPR in an interview.

The approval of the CRISPR gene-editing treatment was also welcomed by Victoria Gray, a Forest, Miss., a sickle cell patient who was the first person to receive it in the U.S. NPR has had exclusive access to chronicle her experience since she was treated in 2019.

Another concern is whether sufficient research had been done to spot "off-target" effects of the treatment — unintended editing errors that missed their mark in the DNA and that could potentially cause long-term health problems.

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