Courtesy of ARIAD Pharmaceuticals

In the gene therapy system devised by Penn and ARIAD researchers, the adeno-associated virus is injected into the body to deliver the desired gene (top left), and a drug in pill form is given orally to activate gene expression (top right). Researchers demonstrated a strong correlation between the amount of drug administered and the resulting increase in the amount of protein produced (center).
Scientists attempting to devise successful, efficient gene therapy systems face three major challenges: transference, persistence, and regulation. First, they must deliver the desired gene into target cells and ensure that it remains in those cells for an extended period of time. They must then, in many cases, regulate that gene so that it's only active when needed. Two recent papers, one in the Jan. 1 issue of Science1 and another in the Jan. 19 issue of Proceedings of the National Academy of...

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