Gene therapy is the use of DNA as a pharmaceutical agent to treat disease. It derives its name from the idea that DNA can be used to supplement or alter genes within an individual's cells as a therapy to treat disease. The most common form of gene therapy involves using DNA that encodes a functional, therapeutic gene in order to replace a mutated gene. Other forms involve directly correcting a mutation, or using DNA that encodes a therapeutic protein drug (rather than a natural human gene) to provide treatment. In gene therapy, DNA that encodes a therapeutic protein is packaged within a "vector", which is used to get the DNA inside cells within the body. Once inside, the DNA becomes expressed by the cell machinery, resulting in the production of therapeutic protein, which in turn treats the patient's disease.
Gene therapy was first conceptualized in 1972, with the authors urging caution before commencing gene therapy studies in humans. The first FDA-approved gene therapy experiment in the United States occurred in 1990, when Ashanti DeSilva was treated for ADA-SCID. Since then, over 1,700 clinical trials have been conducted using a number of techniques for gene therapy.
Scientists at the University of Michigan have shown that gene therapy can be used to successfully stop the development of periodontal disease, the leading cause of tooth loss in adults.
The findings will be published online Dec 11 in advance of print publication in Gene Therapy.
Using gene transfer to treat life threatening conditions is not new, but the U-M group is the first known to use the gene delivery approach to show potential in treating chronic conditions such as periodontal disease, said William Giannobile, professor at the U-M School of Dentistry and principal investigator on the study.
"Gene therapy has not been used in non-life threatening disease. (Periodontal disease) is more disabling than life threatening," said Giannobile, who also directs the Michigan Center for Oral Health Research and has an appointment in the U-M College of Engineering. "This is so important because the next wave of improving medical therapeutics goes beyond saving life, and moves forward to improving the quality of life."
The preclinical study offers was a collaboration with the Seattle-based biotechnology company Targeted Genetics. In July, Targeted Genetics released human trial results that showed the same gene therapy approach used to stop periodontal disease had positive affects in human patients with rheumatoid arthritis, another chronic, non-life threatening, disabling condition. The company tested 127 human subjects and showed a 30 percent improvement in pain relief, and gain of function, among other enhancements using the gene treatment.
