Researchers at the American Society of Gene Therapy Meeting in St. Louis announced that by using imaging technologies, they are able to successfully trace the delivery of DNA nanoparticles and the extent of gene transfer in the lungs of cystic fibrosis (CF) animals.
The study represents an important step in developing gene therapy for cystic fibrosis and other serious lung diseases.
Assem Ziady, Ph.D., assistant professor of pediatrics at the Case Western Reserve University School of Medicine, presented the results. He is conducting studies of a promising non-viral DNA nanoparticle technology that may prove to be effective in treating numerous human diseases. For this study, he collaborated with Zhenghong Lee, Ph.D., assistant professor of radiology and an expert in imaging.
In the study, Ziady and colleagues administered to CF mice DNA nanoparticles encoding a gene for an enzyme that produces light when exposed to a particular chemical. Later, the researchers could then use the emitted light to see in real time where the DNA nanoparticles had delivered the gene for expression in the lungs of the mice.
"Development of these real-time imaging modalities has allowed us to better assess the localization and site of activity of our gene transfer complex formulations," said Ziady. "Understanding gene transfer in animals is important in developing nucleic acid-based therapies to treat serious lung diseases such as cystic fibrosis."
The DNA nanoparticle technology is licensed to and being developed by Cleveland-based Copernicus Therapeutics, Inc. "Collaborations with scientists such as Dr. Ziady enable us to better develop nucleic acid therapeutics for different parts of the body and for multiple diseases," said Mark J. Cooper, M.D., senior vice president of Science and Medical Affairs of Copernicus. "Our lead program, which already has had a successful clinical trial, is development of a therapy for the lung complications of cystic fibrosis."