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Cystic Fibrosis – RNA interference used to silence Protein and correcting Chloride Transport Errors

June 30th, 2006

Cystic Fibrosis – RNA interference used to silence Protein and correcting Chloride Transport Errors


Cystic Fibrosis is a genetic disorder that causes the body to ineffectively move chloride in and out of cells.  Researchers from Johns Hopkins Children’s Center have been able to isolate the overactive protein that causes the transport problem.  They further developed an RNA inference to silence the overproduced protein which made the cells return to normal.  These are preliminary studies and it would take years to develop a treatment for people.  The report was first published in the June 23rd issue of the Journal of Biological Chemistry.


“The hope is that these findings will be used to design therapies and drugs that go beyond symptom management and actually restore normal cell function to prevent CF,” said senior investigator Pamela Zeitlin, M.D., a pulmonologist at the Children’s Center.

The researchers discovered a protein called VCP/pr97 which is a protein containing valosin.  The protein overpowers and kills a chloride transporter inside the cells.  Most cystic fibrosis patients have a problem of the VCP/pr97 protein, which blocks the chloride transport.  When the researchers used RNA interference, which silenced or quieted the protein, it allowed the cells to restore the transport of chloride going in and out of the cell.

If chloride does not move in and out of the cells, there is a buildup of thick and sticky mucous that can be found in the pancreas, and the lungs.  This build up can cause malnutrition and chronic lung infections and even lung damage. The researcher’s goal is to correct the chloride transport mechanism to alleviate all symptoms of cystic fibrosis.


In everyone’s body, there is a built-in quality check system inside cells, called ERAD, which stands for endoplasmic reticulum-associated degradation.  The cells will detect chemically any defects in a protein. The ERAD system sends orders to destroy and dispose of these defective proteins.  The proteasome is the location where the cells dispose of the defective proteins.

Cystic fibrosis people have genetic defects in a protein called CFTR, which stands for cystic fibrosis transmembrane regulator. The ERAD system in cystic fibrosis people have been disturbed which makes it so that the cells are not able to successfully seek out and destroy the defective CFTR protein.

“We were able to confirm that to get rid of the defective CFTR protein, cells deploy VCP/p97 protein, which latches onto the damaged CFTR and sends it to the proteasome for destruction,” Zeitlin says. “Using RNA interference, which basically works by silencing the expression of genes or proteins, we homed in on VCP and blocked its production. That let the defective CFTR to successfully sneak past the quality control and race up to the surface.”


“Targeting VCP, we were able to achieve two things at once -- restoring chloride channel function and curbing inflammation” said co-author Neeraj Vij, Ph.D., a postdoctoral fellow at the Children’s Center. “Inhibiting specific sites in VCP can lead to the development of CF drugs.”

“The goal is to develop small molecules that disrupt the binding between the VC protein and CFTR, much like tiny guided missiles that take out portions of this rampant VC protein before it latches onto CFTR,” Zeitlin said.

While the RNA interference treatment for cystic fibrosis is in its very earliest stages, there is great potential for developing a successful RNA interference treatment in the years to come.  Once the treatment finally make it to market (which could be many years), it could alleviate the detrimental effects of this genetic disorder.  It is estimated that cystic fibrosis effects every one out of every 3,300 live births.

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Nicole Wilson
Best Syndication

Books on Cystic Fibrosis

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Copyright 2005 Best Syndication                   Last Updated Saturday, July 10, 2010 09:47 PM