Gelsinger had a severe gene disorder controlled by drugs and diet but was relatively healthy when he began the therapy. In addition, the results could delay experiments that use viruses to transport the corrective genes into the body - currently the most common treatment method. People say, 'I have a disease and I want gene therapy. Gelsinger died Sept. 17, four days after researchers injected the modified cold virus and engineered genes into an artery leading into his liver. He suffered from ornithine transcarbamylase deficiency, a genetic disorder that leaves the body unable to produce enzymes that remove ammonia from the body.
Ammonia is a byproduct of protein processing.
Most victims of the rare disorder die as infants. Gelsinger had a mild form that he kept in check with medications and low-protein foods. Researchers said they still do not understand precisely why Gelsinger died. The preliminary investigation suggests that the cold virus should not be injected into the bloodstream or liver the way it was done in Gelsinger's case, Wilson said. But it has caused no problems in some other patients.