Researchers analysed thousands of laboratory-made plasmids and discovered that nearly half of them had defects, raising questions of experimental reproducibility.
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On Wednesday, Kendric Cromer, a 12-year-old boy from a suburb of Washington, became the first person in the world with sickle cell disease to begin a commercially approved gene therapy that may cure the condition.
For the estimated 20,000 people with sickle cell in the United States who qualify for the treatment, the start of Kendric’s monthslong medical journey may offer hope. But it also signals the difficulties patients face as they seek a pair of new sickle cell treatments.
For a lucky few, like Kendric, the treatment could make possible lives they have longed for. A solemn and shy adolescent, he had learned that ordinary activities — riding a bike, going outside on a cold day, playing soccer — could bring on episodes of searing pain.
“Sickle cell always steals my dreams and interrupts all the things I want to do,” he said. Now he feels as if he has a chance for a normal life.
Near the end of last year, the Food and Drug Administration gave two companies authorization to sell gene therapy to people with sickle...