Rogue Protein May Be Key to Cystic Fibrosis

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FRIDAY, July 7 (HealthDay News) -- U.S. researchers say they've identified and controlled an overactive protein that may be a key player in cystic fibrosis (CF).

CF is a genetic disorder that interferes with the body's ability to transport chloride in and out of cells.

According to researchers at Johns Hopkins Children's Center, Baltimore, the newly identified protein, called VCP/pr97, kills a chloride transporter in the cells of most CF patients. This inability to transport chloride results in a dangerous buildup of thick, sticky mucous in several organs, including the pancreas and lungs. That can lead to the malnutrition, chronic lung infection and lung damage so often seen in CF patients.

In this test-tube study, the researchers used a tool called RNA interference to successfully intercept signals sent out by VCP/pr97. In doing so, they were able to prevent cell damage caused by the protein. The findings showed promise in restoring cells to normal status in CF patients, the team said.

"The hope is that these findings will be used to design therapies and drugs that go beyond symptoms management and actually restore normal cell function to prevent CF," senior investigator Dr. Pamela Zeitlin, a pulmonologist at Johns Hopkins Children's Center, said in a prepared statement.

However, it will take years before any treatments based on this research are developed or tested on animals or humans, she noted.

The study was published in the June 23 issue of the Journal of Biological Chemistry.

More information

The March of Dimes has more about cystic fibrosis.

SOURCE: Johns Hopkins Children's Center, news release, June 29, 2006