University of North Carolina at Chapel Hill's Gene Therapy Department

The University of North Carolina School of Medicine created the Gene Therapy Center in 1996 with the goal of merging molecular genetics research with healthcare delivery. The Gene Therapy Center provides important resources to academic investigators through two core facilities created to support preclinical and clinical gene therapy studies. These facilities, the Vector Core and the Human Applications Laboratories, were created to ensure that investigators would have promising gene vectors available in the quality and quantities needed for preclinical or clinical studies. Research in the laboratory has centered on the molecular biology of adeno-associated virus (AAV) in order to exploit the unique features of this virus to develop an efficient viral vector system for use in human gene therapy. 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. The ultimate goal of the Gene Therapy Center is to facilitate the progression and translation of gene therapy research from the laboratory bench into Phase I clinical trials for the treatment of human disease. Click here to learn more about our gene therapy research.

June 2008 - Jude Samulski, Director, has received the inaugural Oustanding Achievement Award given by the American Society of Gene Therapy. The award recognizes an active member of the society who has achieved a pioneering research success, a specific high-impact accomplishment or a lifetime of significant contributions to the field of gene therapy. Click here for the official press release.

March 2006 - The first clinical trial for Duchenne Muscular Dystrophy begins, utilizing AAV vectors developed and produced in Dr. Samulski's laboratory. Click here for more information.

March 2004 - Drs. Stilwell and Samulski published a breakthrough study on the safety of AAV virion shells. They found that viral capsids may influence gene expression independent from their packaged DNA. AAV and adenovirus were compared using DNA microarrays for their effects on cellular gene expression. AAV was found to have minimal impact on gene expression in the cell, while adenovirus activated the expression of genes associated with toxicity and the immune response. These findings indicate that AAV may be a safer and more effective form of transduction. Click here for more information about this study.