Since the death last September of an Arizona teenager, the first person to die of gene therapy, many gene therapists are looking to gutless adenovirus vectors to mend the reputation of adenovirus-based gene therapy. Gutless vectors are named for their lack of adenovirus genes. If, as expected, they prove safer and provide longer lasting therapeutic gene expression, adenovirus vectors like the one that killed Jesse Gelsinger will likely be phased out for most diseases.
Why Gelsinger died remains unknown. But multiple investigations of both his clinical trial and gene therapy regulation have been launched. The Food and Drug Administration, after finding numerous errors in the Gelsinger trial, in January suspended all seven trials in progress where he was treated, the Institute for Human Gene Therapy in Philadelphia. On the heels of that event is publication in February of long-anticipated work on gutless vectors1 by Volker Sandig, Thomas Caskey, and their ...
