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Rewiring the Genome: The Future of Medicine Gets Personal
Fifteen years ago, the promise of gene therapy was dealt a stunning blow by the widely reported death of eighteen-year-old Jesse Gelsinger during a clinical trial. Researchers were attempting to cure Gelsinger and other patients suffering from a rare condition called ornithine transcarbamylase deficiency, which leaves people unable to process nitrogen in their blood—making protein-rich foods potentially deadly. To treat Gelsinger, scientists injected him with a virus engineered to include a functional version of the gene that was faulty in OTCD patients. But within 48 hours, Gelsinger’s condition dived dramatically; his body swelled, his skin and eyes yellowed, showing signs of liver damage, and his fever spiked dangerously high. Four days after injection, doctors found Gelsinger had no hope of recovery, and his family decided to take him off of life support.
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