The Recombinant DNA Advisory Committee (RAC) meeting in Washington, D.C., Dec. 8-10 delved into every aspect of Jesse Gelsinger's death. Gelsinger, a teenager from Arizona, had ornithine transcarbamylase (OTC) deficiency, a liver disorder in which the body cannot eliminate ammonia through the urea cycle. He died four days after receiving an experimental gene therapy drug during a University of Pennsylvania clinical trial.
After hearing hours of clinical data from researchers involved in the trial and from Food and Drug Administration officials, RAC heard what the public had to say. Parents of children suffering from rare genetic disorders pleaded passionately in favor of gene therapy clinical trials. Paul Geisinger, Jesse's father, even supported the technology. Claudia Mickelson, chair of the 15-member group that reviews safety issues involved with gene therapy experiments, assured them that the purpose of the meeting was not to halt gene therapy, but to ensure the safety of ...
