The UNC Gene Therapy Center provides important resources to academic investigators through two A crucial feature of the UNC Gene Therapy Center is the creation of two core facilities, which were created to support preclinical and clinical gene therapy studies. The core facilities available to gene therapy investigators include the Vector Core Laboratory and the Human Applications Laboratory. The unique features that make this virus attractive for human gene therapy include the facts that it is able to insert its genome locus-specifically into human chromosome 19 and that it has never been identified as a causative agent of human disease. Continued efforts in understanding the mechanism of viral replication and integration for both wild-type and recombinant AAV are being pursued in order to create more efficient gene transfer vectors. Typically, curing cellular dysfunction is synonymous with curing a disease that involves a whole organ.
Gene transfer offers exciting prospects for specific therapy of human diseases.
The specificity of gene transfer therapies stems from the fact that usually only one gene is transferred for therapeutic purposes, and consequently, only a single new protein is introduced into the cell. The potential application of gene transfer therapies to medical diseases is limited only by our knowledge of the molecular basis for certain diseases. It is highly likely that in these latter diseases, highly efficient, novel ways of treating these diseases can be achieved. It is clear that delivery of the proper specific protein may slow the growth of cancers, and delivery of proteins that modulate the inflammatory response in asthmatic airways could be a successful therapeutic approach. Gene therapy must be brought to the patient.