- Molecular Therapeutics
- UNC-Chapel Hill
- 7119 Thurston-Bowles
Area of Interest
Our research centers around developing viral vectors for gene delivery with the long term objective of using these reagents for cancer gene therapy. To date, we have been successful in establishing both parvo and adenovirus as efficient delivery systems for both in vitro and in vivo gene delivery. Vectors carrying genes that would augment chemotherapy (TK) as well as anti-oncogenes have been utilized in these vectors. Testing in tumor bearing animals for effective gene delivery and reduction of tumor has been established through collaborative efforts. We anticipate eventual development of this strategy for clinical intervention. Below are a list of selective publications that continue to demonstrate our ability to improve on viral vectors and efficient gene delivery.
Awards and Honors
In October 2008, Dr. Samulski and the Gene Therapy Center were awarded a 5 year Senator Paul B. Wellstone Muscular Dystrophy Cooperative Research Center award. . There are only 6 Wellstone Centers in the US. Our Center will pursue 3 projects - 1) A clinical trial to establish and ensure safety of gene delivery to large muscular groups, 2) Investigate gene delivery methods to determine the optimal method for delivery to large muscle groups, 3) Optimize the "gene delivery vehicle" for treating muscle disorders.
In June 2008, Dr. Samulski 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
In March 2003, Drs. Stilwell and Samulski published a breakthrough study in Molecular Therapy 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.