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.

Dr. Steven Gray and his wife Christy attended the Hope and Love Ball in Albany, NY on February 12. This was a fundraiser for Hannah’s Hope Fund, an organization supporting Dr. Gray’s research at the UNC Gene Therapy Center to develop a treatment for Giant Axonal Neuropathy (GAN). Among the 350 attendees was Doris Buffet, sister of billionaire Warren Buffet and founder of the Sunshine Lady Foundation, who had previously donated $500,000 to Hannah’s Hope Fund. Also in attendance was Hannah, the 6-year-old namesake for Hannah’s Hope. Drs. Steven Gray and Maria Escolar (UNC Gene Therapy Center) are spearheading an international effort to study and treat GAN. GAN is an orphan disease caused by a loss of the gigaxonin gene, and it is characterized by motor and sensory system degeneration that is terminal by the 3^rd decade of life.

October 2010 - Richard J. Samulski and the Gene Therapy Center are making exciting findings on Duchenne muscular dystrophy (DMD). Click here to learn more.

October 2009 - Gene Therapy PI You Tube! Joe Kornegay was interviewed at the American College of Veterinary Internal Medicine (ACVIM) in Seattle last June. The context within ACVIM is that their work is an example of "One Medicine" or "Comparative Medicine" intended to advance the understanding of diseases in both humans and animals.

Cure for Duchenne Muscular Dystrophy Lies in Paws of Man's Best Friend

Please join UNC's Air Force ROTC on Thursday, October 15 for a Push-Up-A-Thon to help raise money for MD research. The event will be held at 4 pm in front of The Center for Dramatic Art. All proceeds will go to the Senator Paul Wellstone Muscular Dystrophy Research Center to support MD research. If you'd like to help sponsor this event, please send an email to rnathani@email.unc.edu.

August 2009 - The UNC Gene Therapy Center was featured in the August 30 edition of Parade magazine. Parade published the article in anticipation of the annual Jerry Lewis Muscular Dystrophy Association Telethon, which commences Sunday, September 6, 2009.

May 2009 - Dr. Samulski was elected vice president of the American Society of Gene and Cell Therapy. This is a three year cycle - one year as Vice President (2009-2010), then one year as President-Elect (2010-2011), and a third year as President (2011-2012). He will be President during the 15th Annual Meeting of ASGCT taking place in Philadelphia, PA May 16 - 20, 2012. http://www.med.unc.edu/pharm/news/faculty/samulski-presElect-ASCGT.html

March 2009 - The UNC Senator Paul D. Wellstone Center of Excellence for Muscular Dystrophy Research held its inauguration event on March 2, 2009. Distinguished guests included Rep. David Price and Chancellor Holden Thorpe.

October 2008 - UNC Gene Therapy Center has been awarded a five-year, $7 million grant to establish a muscular dystrophy research center named in honor of the late Sen. Paul D. Wellstone of Minnesota, who earned his undergraduate and doctoral degrees at UNC. Click here for the official press release

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.