Defective signaling pathway sheds light on cystic fibrosis

Contact: Debra Kain
ddkain@ucsd.edu
619-543-6163
University of California - San Diego
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 contributes to disease severity. In the study, published in the journal Nature Medicine, the researchers report that defective signaling for a protein called the peroxisome proliferator-activated receptor-ã (PPAR-ã) accounts for a portion of disease symptoms in cystic fibrosis, and that correction of the defective pathway reduces symptoms of the disease in mice.

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Research project yields better understanding of the defective protein that causes cystic fibrosis

Contact: Angela Hopp
ahopp@asbmb.org
301-634-7389
American Society for Biochemistry and Molecular Biology Read more

Scripps research team restores some function to cells from cystic fibrosis patients

Contact: Keith McKeown
kmckeown@scripps.edu
858-784-8134
Scripps Research Institute Read more

Sodium channel blocker shows promise as a potential treatment for cystic fibrosis

Cystic fibrosis patients may benefit from a new therapy that increases airway hydration, preventing the buildup of mucous, which is a key factor in the disease, according to researchers at Parion Sciences in Durham, N.C.

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Gene therapy treatment For Cystic fibrosis sufferers in the works

Scientists found that a laboratory-created “super” virus could be used to deliver a healthy version of a gene into cells affected by cystic fibrosis, ridding them of the disease.

The discovery is the latest boost for a technique called gene therapy, which scientists hope can attack diseases at their root.

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Possible preventative treatment for cystic fibrosis lung disease tested

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Viral DNA in bacterial genome could hold key to new cystic fibrosis treatments

The bacterium Pseudomonas aeruginosa is well known for its environmental versatility, ability to cause infection in humans, and its capacity to resist antibiotics. P. aeruginosa is the most common cause of persistent and fatal lung infections in cystic fibrosis patients. In a study published online today in Genome Research (www.genome.org), researchers have used genomic techniques to study a particularly virulent strain of P. aeruginosa, uncovering genetic clues to its success that will aid in the design of novel therapeutic strategies.

The Liverpool Epidemic Strain, the most common strain of P. aeruginosa infecting cystic fibrosis patients in the United Kingdom, is characterized by its particular aggressiveness and virulence. Though approximately 90% of the P. aeruginosa genome is shared between different strains, a team of scientist led by Dr. Craig Winstanley of the University of Liverpool set out to investigate the unique genomic features of the Liverpool strain. ”We used genome sequencing to reveal the secrets of the other 10% of the genome, which is likely to include genes contributing to the success of this particular strain,” said Winstanley.

The research group found that many of the genes specific to the Liverpool Epidemic Strain are positioned in clusters, some of which are prophages. A prophage is a set of viral genes that became integrated into the DNA of bacteria infected by a bacterial virus. They then engineered mutations in prophages of the Liverpool strain and tested the pathogenicity of the mutant bacteria in a rat model of chronic lung infection. “We have shown that mutations within these novel prophages and genomic islands can prevent the strain from establishing infections,” Winstanley described. ”This indicates that bacterial viruses may contribute to the ability of bacterial pathogens to adapt to specific environments and to the emergence of particularly successful epidemic bacterial strains.”

Winstanley also explained that this work is especially important in light of the nature of P. aeruginosa lung infections that afflict cystic fibrosis patients. Once an infection has been established, antibiotic therapy is unable to eradicate the bacteria from the lungs. Since antibiotics can be of limited use, the genomic properties of the Liverpool Epidemic Strain characterized in this study will aid in the development of novel strategies for circumventing ineffective antibiotic treatments by preventing infection altogether.


Scientists from the University of Liverpool (Liverpool, UK), Simon Fraser University (Burnaby, BC), Laval University (Quebec City, QC), The Wellcome Trust Sanger Institute (Hinxton, UK), and the University of British Columbia (Vancouver, BC) contributed to this study.

This work was supported by the Wellcome Trust, the Canadian Institutes of Health Research, the Canadian Cystic Fibrosis Foundation, the US Cystic Fibrosis Foundation, the Michael Smith Foundation for Health Research, the Big Lottery Fund, and the UK Cystic Fibrosis Trust.

New Test Promises Quicker, More Accurate Evaluation for Cystic Fibrosis Patients

Simple blood test could improve patient care and speed drug trials

Researchers at National Jewish Health have identified a simple gene-based blood test that more accurately and quickly measures cystic fibrosis patients response to therapy than current tests. The test, a measure of inflammatory gene expression, could improve patient care and help clear a backlog of promising medications now hung up in clinical trials. The researchers are publishing the results of a small proof-of-principal trial in the November 1, 2008, issue of American Journal of Respiratory and Critical Care Medicine .

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New drug hope for cystic fibrosis patients

A new drug therapy may represent a tremendous step forward in the treatment of some 70,000 cystic fibrosis (CF) patients worldwide, Dr David Sheppard from the University of Bristol told an audience at the BA Festival of Science in Liverpool today [9 September].

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Clearing the airways in cystic fibrosis

Research in the FASEB Journal shows that engineered proteins can ‘bypass’ the genetic defect in cystic fibrosis

By manipulating the machinery used by our cells for quality control, researchers from the University of Pittsburgh have found a way to restore the function of cystic fibrosis (CF) airway cells. This could significantly reduce the sticky mucus that plugs the lungs of CF patients, which leads to antibiotic-resistant infections and untimely death. The study, appearing in the September 2008 print issue of The FASEB Journal (http://www.fasebj.org), is significant because it shows a new way to manipulate the cellular quality controls of all sorts of proteins which play a role in conditions ranging from aging to cancer.

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