She donned a paper gown, bonnet, booties and gloves, and entered the supersterile environment of the human application laboratory - HAL - of the Institute for Human Gene Therapy.
It was there, in a sci-fi world of hooded lab tables, freezers, petri dishes and microscopes, that Grossman would use the viruses to produce what hopefully would be the first gene therapy drug effective against a major genetic disease, cystic fibrosis.
If all went well, the drug would make medical history.
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It was the spring of 1994. James M. Wilson, the principal investigator in the gene therapy study and director of the institute, had great hopes for the
drug Grossman was about to produce.
He thought the chances were good that it would give doctors a way to control cystic fibrosis with treatments given every couple of months. Learning how to do this would provide treatments, perhaps cures, for scores of other diseases as varied as cancer, heart disease and AIDS.
Called vectors, gene therapy drugs are radically different from conventional medicine. Most are viruses that have been bioengineered to carry genes into a patient's cells. The genes then force cells to make proteins that fight disease or compensate for genetic defects.
Wilson had reason for optimism. A first-generation version of this virus, Vector I, had looked promising in human trials. It was an adenovirus, which normally causes colds, but it had been disabled. Now Wilson's lab had developed a second-generation virus that appeared even better. He had so much faith in it that he abruptly stopped testing Vector I on patients.
Vector II was less likely to produce dangerous immune reactions at high doses. And it worked for at least a month, twice as long as Vector I.
Wilson hoped Vector II would do even better in humans.
No one was following the trials more eagerly than cystic fibrosis patients who knew that research was advancing with remarkable speed.
