This story is part of a series on the current progression in Regenerative Medicine. In 1999, I defined regenerative medicine as the collection of interventions that restore tissues and organs damaged by disease, injured by trauma, or worn by time to normal function. I include a full spectrum of chemical, gene, and protein-based medicines, cell-based therapies, and biomechanical interventions that achieve that goal.
The 1990s were a crucible for gene therapy, with efforts around the globe pushing boundaries, driven by the audacious vision of curing diseases at their root—the genetic level. This decade saw clinical trials that delivered the first definitive evidence that gene therapy was more than just a science fiction fable.. The two girls had a rare and life-threatening disorder called severe combined immunodeficiency , which was caused by a genetic mutation.
In 1999, Gelsinger, who had a genetic liver disease called ornithine transcarbamylase deficiency, participated in a gene therapy trial at the University of Pennsylvania. The trial aimed to develop a treatment for infants with severe forms of the disease. Despite having a milder form of the disorder, Gelsinger volunteered for the trial and was given an injection of an adenoviral vector carrying a corrected gene.
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