GeneMedicine preclinical data

The Houston company, writing in the Proceedings of the National Academy of Sciences, showed that a gene for tyrosine hydroxylase combined with a gene switch could be controlled by giving an oral steroid. Expression of the introduced gene in rats was increased 10-fold compared to controls. The doses of steroid used to activate the switch construct are orders of magnitude too low to produce steroid side effects, the researchers showed.

The technology is licensed exclusively from Baylor College of Medicine. The switch is a modified steroid receptor that is activated only by certain synthetic steroids, including the abortion inducer RU 486. The steroid activates the receptor to bind to a gene sequence incorporated within the introduced gene, turning on expression of the gene. ...