| From the Editor's Desk
The Era of One-Shot, Multimillion-Dollar Genetic Cures Is Here Some of Steven Pipe’s hemophilia patients consider themselves cured. In a trial Pipe led from 2018 to 2021, they received a one-time gene therapy meant to override a DNA mutation that causes spontaneous bleeding episodes, some of them severe and life-threatening. Unlike most drugs, which relieve symptoms, gene therapy addresses the underlying cause of a disease. Thanks to the treatment, they haven’t had to worry about serious bleeding for years.
“They don’t have to think about their hemophilia anymore,” says Pipe, a hematologist at the University of Michigan Health System. “For all intents and purposes, this looks like a cure.”
Continued here
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