One step closer to a drug treatment for cystic fibrosis

Oct 12, 2010

A University of Missouri researcher believes his latest work moves scientists closer to a cure for cystic fibrosis, one of the world's most common fatal genetic diseases.

The has published findings by Tzyh-Chang Hwang, a professor in the School of Medicine's Department of Medical Pharmacology and Physiology and the Dalton Cardiovascular Research Center. The publication has been recognized as the "paper of the week" for the journal, meaning Hwang's work is considered to be in the top 1 percent of papers reviewed annually in terms of significance and overall importance.

Hwang's work focuses on the two most common among approximately 1,500 mutations found in patients with . These two mutations cause specific chloride channels in the cell, known as the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) chloride channels, to malfunction. This ultimately leads to repeated pneumonia, the primary cause of most deaths associated with cystic fibrosis.

"The normal function of a cell is to pass across the at a very fast speed," Hwang said. "We know some signaling molecules elicit this reaction, much like a hand signals an automatic water faucet to dispense water. But in the case of cystic fibrosis, that signal is no longer detected by the mutated channel protein. Through some mechanisms we still don't quite understand, malfunction of this channel protein eventually leads to in the lung, which is believed to be responsible for the most severe symptoms of cystic fibrosis."

The most recent study found that manipulating the sensor of the channel protein can significantly rectify the malfunction of the mutated channel, thus opening the door to a drug design that may eventually be a "real cure," Hwang said.

"We could help a lot of patients if we can utilize the power of computer simulations and structure-based drug design to discover new therapeutical reagents for cystic fibrosis, but it's very expensive to do this kind of research in an academic institute," Hwang said.

Explore further: Dead feeder cells support stem cell growth

Related Stories

At last, a living model for an important body channel

Oct 07, 2010

(PhysOrg.com) -- Ion channels provide a way for key molecules to cross into cells, are the means for many swift physical reactions and regulate the movement of fluid across internal cavities in our bodies. When chloride ions ...

Defective signaling pathway sheds light on cystic fibrosis

Feb 14, 2010

In a study that could lead to new therapeutic targets for patients with the cystic fibrosis, a research team from the University of California, San Diego School of Medicine has identified a defective signaling pathway that ...

Recommended for you

Dead feeder cells support stem cell growth

Apr 24, 2015

Stem cells naturally cling to feeder cells as they grow in petri dishes. Scientists have thought for years that this attachment occurs because feeder cells serve as a support system, providing stems cells ...

Improving accuracy in genome editing

Apr 23, 2015

Imagine a day when scientists are able to alter the DNA of organisms in the lab in the search for answers to a host of questions. Or imagine a day when doctors treat genetic disorders by administering drugs ...

Drug research enhanced by fragment screening libraries

Apr 22, 2015

Generation of fragment screening libraries could enhance the analysis and application of natural products for medicinal chemistry and drug discovery, according to Griffith University's Professor Ronald Quinn.

Decoding the cell's genetic filing system

Apr 22, 2015

A fully extended strand of human DNA measures about five feet in length. Yet it occupies a space just one-tenth of a cell by wrapping itself around histones—spool-like proteins—to form a dense hub of ...

User comments : 0

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.