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Novel gene therapy approach in 1st human trials

CLEVELAND, April 2, 2002: A 33-year old cystic fibrosis patient underwent the first gene therapy trial today using a novel approach to fight a deadly genetic disease that affects more than 30,000 Americans. The researchers include a team from The Research Institute of University Hospitals of Cleveland, its Rainbow Babies & Childrens Hospital, Case Western Reserve University School of Medicine, and Copernicus Therapeutics Inc., developers of the novel gene transfer system.

Over the last decade, there have been various attempts to transfer healthy genes into the cells of the airways of cystic fibrosis patients, explains primary investigator Michael W. Konstan, MD, Director of the LeRoy Matthews Cystic Fibrosis Center at Rainbow Babies & Childrens Hospital, and associate professor of pediatrics at CWRU School of Medicine. Most of these trials have involved the use of a viral vector, attaching the healthy gene to a virus that will invade cells. Unfortunately, the virus itself has caused troublesome inflammation in the study patients.

For more than a decade, UHC/CWRU researchers have explored alternative methods to viral vectors. Their work led to a patented approach produced by the Cleveland-based biotechnology firm, Copernicus Therapeutics, Inc. They developed a way to compact or tightly bind strands of DNA so that it is tiny enough to pass into the cell and then into the nucleus. Once inside a cell, researchers hope that the DNA will produce the normal version of the protein needed by cystic fibrosis patients.

Thirteen years ago, scientists discovered the cystic fibrosis (CF) gene. This defective gene upsets a delicate salt/water balance in the lungs. At the crux of the process is a protein, produced by the CF gene, which controls the flow of salt and water in and out of cells. In CF patients, this protein does not operate normally in the cells that line the airways. In turn, the airways accumulate thick and sticky mucus. Bacteria
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Contact: Eileen Korey
eileen.korey@uhhs.com
216-844-3825
University Hospitals of Cleveland
2-Apr-2002


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