One-Step, Two-Step Regulation of Therapeutic Genes

In February The Scientist ran a timely article by Eugene Russo discussing the new and exciting advances in the regulation and targeting of genes for gene therapy procedures.1 The elegant studies from research groups led by James Wilson, Michael Gilman, and Bert O'Malley showed that it is possible to deliver a putative therapeutic gene to specific tissues, in a dormant state, and turn the gene on and off at will by orally administering a second drug. The second drug orchestrates the formation of

Keith Webster
Apr 25, 1999

In February The Scientist ran a timely article by Eugene Russo discussing the new and exciting advances in the regulation and targeting of genes for gene therapy procedures.1 The elegant studies from research groups led by James Wilson, Michael Gilman, and Bert O'Malley showed that it is possible to deliver a putative therapeutic gene to specific tissues, in a dormant state, and turn the gene on and off at will by orally administering a second drug. The second drug orchestrates the formation of an active transcription factor complex on the promoter of the transferred gene.2,3 Tests were carried out in both mice and rhesus monkeys over periods of up to one year. The groups used different viral vectors, regulatory systems, activator drugs, and therapeutic genes, but the kinetics, magnitude, and duration of transferred gene expression were comparable in the two studies, and both studies demonstrated physiological impact; in...