Dr. Samulski and members of the Gene Therapy Center have recently developed novel viral vectors with targeting capability. These reagents will be of significant value when transducing specific cell types such as hepatocytes.
One of the great aspirations of gene therapy is to eventually develop technology which will provide a feasible approach to correcting genetic defects and combating infectious diseases. The laboratory is engaged in studying the molecular biology of the defective human parvovirus adeno-associated virus (AAV) in hopes of developing a safe, efficient viral vector for human gene therapy. Ongoing research is revealing that this nonpathogenic human virus is now accessible for utilization as a vector. We have developed a packaging system which allows for efficient encapsidation of foreign genes into AAV virions. We have also indentified the essential cis-acting sequencing required for efficient integration into host cell DNA. Finally, we have characterized this integration step and uncovered the exciting result of site-specific integration. this last observation clearly sets AAV apart as a eucaryotic viral vector and its potential for gene therapy in humans. Our continued efforts to understand and manipulate this virus as a vector has required a multi-faceted approach from basic virology in vivo to in vitro virus replication, integration, and packaging.