Therapy Researchers
Sidebar: Summary Table of Vectors
According to the Pasteur Institute, a nonprofit research organization in Paris, 216 gene therapy protocols with human patients-treating diseases such as cystic fibrosis, cancer, and AIDS-are under way worldwide.
In the vast majority, therapeutic genes are packed into disarmed viruses, which shuttle the genetic payload into a cell's chromosomes.
These viral vectors require the expertise of virologists, biochemists, and molecular biologists, among others. Meanwhile, nonviral vectors-for example, synthetic molecules that tote therapeutic genes-call for biophysicists and analytical chemists.
Gene therapy researchers agree that finding-or creating-viable vectors is of utmost importance to patients and investigators. "There isn't much future to gene therapy without viable vectors," remarks Didier Trono, head of infectious diseases research at the Salk Institute for Biological Studies in La Jolla, Calif. "This is where work needs to be done."
Four types of vectors share the gene therapy spotlight: retroviruses, such as ...