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
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."
About Case Western Reserve University
Case is among the nation's leading research institutions. Founded in 1826 and
shaped by the unique merger of the Case Institute of Technology and Western
Reserve University, Case is distinguished by its strengths in education,
research, service, and experiential learning. Located in Cleveland, Case
offers nationally recognized programs in the Arts and Sciences, Dental Medicine,
Engineering, Law, Management, Medicine, Nursing, and Social Sciences. http://www.case.edu .
About Copernicus Therapeutics, Inc.
Copernicus Therapeutics, Inc., a privately held biotechnology company, is advancing
novel nucleic acid delivery systems with broad applications in human therapeutics.
Additional information about Copernicus is available at http://www.cgsys.com