The new Biostrophin therapy uses a novel combination of advanced technologies, including a miniaturized replacement dystrophin gene and nano delivery technology called biological nanoparticles. Developed from a virus known as adeno-associated virus (AAV), the nanoparticles are engineered specifically to target and carry the "minidystrophin" gene to muscle cells.
The therapy was made possible by the pioneering research in AAV by Dr. Richard Jude Samulski, professor of pharmacology and director of the Gene Therapy Center at UNC, and Dr. Xiao Xiao, a former UNC postdoctoral researcher in Samulski's laboratory now with the University of Pittsburgh Human Gene Therapy Center and associate professor of orthopedic surgery.
Samulski has long pioneered methodologies for making viruses deliver genes. As a graduate student at the University of Florida in the early 1980s, his thesis project was developing the AAV as a vector for therapeutic genes. This work eventually led to isolation of type-2 AAV, which has been used for gene therapy trials in cystic fibrosis and in several other settings, Samulski said.
"It's what we would call the parent virus that everybody started with."
Samulski moved to the University of Pittsburgh in 1986, joining the biology department as an assistant professor with his own laboratory. His first graduate student was Xiao, who had just come from China. Xiao focused on the lab's AAV vector project. "We've continued to have productive collaborations ever since he graduated from my lab," Samulski said.
In 1993, Samulski moved to the University of North Carolina at Chapel Hill, becoming director of UNC's new Gene Therapy Center. Xiao moved first into industry, then to UNC as a postdoctoral researcher in Samulski's gene therapy center. In 1996, the team published a report of the first muscle gene deli
Contact: Stephanie Crayton
University of North Carolina at Chapel Hill