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In its early days, gene therapy often made very sick people even sicker. The advent of safer viral vectors appears to be restoring the promise of this technology.
An international team created a lentivirus vector that carried a functional β-globin gene and used the construct to treat an 18-year-old man who had severe β-thalassemia. In the laboratory, the vector introduced the functional gene into the patient's hematopoietic stem cells (HSCs). After chemotherapy reduced the number of the patient's genetically defective HSCs, the team reinfused his genetically engineered HSCs. During the next several months, the number of circulating red cells with a functional gene rose, as did the patient's level of normal β-globin. Three years out, the pati…