AU Researchers Refining New Gene Therapy-Based Drug Delivery System

A new in vivo drug delivery system that can aid in the treatment of such medical conditions as muscular dystrophy, diabetes, heart disease and muscle-related tumors, is being refined by an Auburn University research team at the College of Veterinary Medicine’s Scott-Ritchey Research Center.

Associate Professor Bruce Smith, and his colleagues Assistant Professor Tatiana Samoylova and Professor Henry Baker, director of the center, have applied for a patent on the new gene therapy-based technology.

Dr. Smith explains that the development is a spinoff from an earlier collaborative project with the University of Alabama in Birmingham.

"In that project, we were searching for a better way to treat muscular dystrophy," he said. "We found that gene therapy could be used to treat individual muscles by injection, but we needed a way to treat the entire skeletal muscle system without injecting every muscle — which would be most unpleasant for the patient."

The AU research team is working to develop a way to administer the treatment intravenously. Dr. Smith explains that the technique uses small peptides, which are chains of seven different amino acids, to target treatments to specific tissues. These targeting peptides are central to successful treatment.

"The research involves examining phage display libraries, collections of more than one billion different peptides attached to a purified virus," Dr. Smith said. "These viruses normally will not bind to a cell, but a few of the peptides enable the virus to bind to muscle tissue."

He says that the process appears to be identifying new artificially created molecular sequences. The research project is now focused on optimizing the ability of these peptides to bind to skeletal muscle tissue by identifying the specific component of the cell to which they attach, and finding peptides that bind to other tissues ? such as brain and tumor cells.

"Once we maximize the interaction of these peptides with their targets, these molecules can be linked to a drug or gene therapy treatment, and the treatment targeted to specific tissues," Dr. Smith added.