The first human trials of gene therapy began in 1990 using a strategy of ex vivo gene therapy. In this approach, the patient cells were harvested and cultivated in the laboratory and incubated with vectors to modify their genes. The cells were then harvested and transplanted back into the patient from whom they derived. The first therapeutic trials utilizing this approach attempted to treat two genetic disorders, including children with an inherited form of immune deficiency as well as children and adults with extremely high levels of serum cholesterol. The first model for in vivo gene therapy was based on an attenuated version of the adenovirus in the treatment of cystic fibrosis.
Adenoviruses have a natural tropism for lungs in that they are associated with respiratory diseases.
Many other types of diseases are currently being investigated as candidates for gene therapy including cardiovascular diseases, infectious diseases such as AIDS, and cancer. The IHGT is the first academic based program dedicated solely to gene therapy. Over the last four years, the Institute has grown and established research programs in virtually all areas of medicine. Over 170 faculty at the University of Pennsylvania participate in Institute sponsored activities. The foundation of the Institute lies in its basic investigation, which is making discoveries necessary for gene therapy to be truly effective in the coming years.