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.
Stephenson J. Studies Illuminate Cause of Fatal Reaction in Gene-Therapy Trial. JAMA. 2001;285(20):2570. doi:10.1001/jama.285.20.2570-JMN0523-3-1