Banff, Alberta—Researchers believe that new findings from animal studies shed light on what caused the devastating immune system reaction that resulted in the first known death of a participant in a gene-therapy trial.
The evidence indicates that the protein coat of the vector—a disarmed adenovirus used to ferry a normal gene into the patient's liver cells—triggered the innate immune response featuring a massive release of cytokines, said James Wilson, MD, PhD, director of the University of Pennsylvania's Institute for Human Gene Therapy.
Wilson, who supervised the trial, presented his findings to researchers gathered here for an American Society for Microbiology–sponsored conference on gene therapy.
The goal of the trial of 18 patients was to assess the safety of various doses of the gene vector. Researchers hoped the gene would correct the metabolic disorder known as ornithine transcarbamylase deficiency (OTC). Jesse Gelsinger, the 18-year-old patient who died . . . [Full Text of this Article]
