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Clearing the airways in cystic fibrosis
August 29, 2008Research 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.
"Our hope is that this work will provide new approaches for recovering function from the protein whose inability to fold and function properly compromises the quality of life and life expectancies of people with CF," said Raymond Frizzell, senior author on the study. The researchers say, however, that it is too early to tell exactly what other conditions this technique will affect.
Most people do not realize that when our bodies produce proteins, they sometimes make mistakes. Similar to what happens on assembly lines, these mistakes are rejected and prevented from being used. In most people with CF, however, defective (i.e., mutant) CFTR proteins made by airway epithelial cells could actually be beneficial if they were allowed to reach their final destination at cell surfaces. To help these mutant proteins (the {DELTA}F508 CF gene mutation) reach cell surfaces, researchers developed a "decoy," made up of only a portion of the complete mutant protein, which is rejected instead. In airway epithelial cells taken from CF patients, the "distracted" airway cells allowed the complete {DELTA}F508 CFTR protein to evade the quality control mechanisms that would have normally destroyed it.
"Since this pivotal discovery of the CF gene nearly 20 years ago, researchers have made more progress toward a cure than they had in thousands of years before," said Gerald Weissmann, MD, Editor-in-Chief of The FASEB Journal. "People with cystic fibrosis now live longer than once ever thought possible, and basic science breakthroughs like this one keep the trend ever upward."
According the U.S. Cystic Fibrosis Foundation, cystic fibrosis affects about 70,000 people worldwide. It is caused by a defective gene and its protein product, which causes the body to produce very thick and sticky mucus. This mucus clogs the lungs and leads to life-threatening lung infections. It also blocks enzymes from the pancreas that needed to break down and absorb food. Sixty years ago, few children with cystic fibrosis lived to attend elementary school. Today, the projected median survival age is 37.
Federation of American Societies for Experimental Biology
Most people do not realize that when our bodies produce proteins, they sometimes make mistakes. Similar to what happens on assembly lines, these mistakes are rejected and prevented from being used. In most people with CF, however, defective (i.e., mutant) CFTR proteins made by airway epithelial cells could actually be beneficial if they were allowed to reach their final destination at cell surfaces. To help these mutant proteins (the {DELTA}F508 CF gene mutation) reach cell surfaces, researchers developed a "decoy," made up of only a portion of the complete mutant protein, which is rejected instead. In airway epithelial cells taken from CF patients, the "distracted" airway cells allowed the complete {DELTA}F508 CFTR protein to evade the quality control mechanisms that would have normally destroyed it.
"Since this pivotal discovery of the CF gene nearly 20 years ago, researchers have made more progress toward a cure than they had in thousands of years before," said Gerald Weissmann, MD, Editor-in-Chief of The FASEB Journal. "People with cystic fibrosis now live longer than once ever thought possible, and basic science breakthroughs like this one keep the trend ever upward."
According the U.S. Cystic Fibrosis Foundation, cystic fibrosis affects about 70,000 people worldwide. It is caused by a defective gene and its protein product, which causes the body to produce very thick and sticky mucus. This mucus clogs the lungs and leads to life-threatening lung infections. It also blocks enzymes from the pancreas that needed to break down and absorb food. Sixty years ago, few children with cystic fibrosis lived to attend elementary school. Today, the projected median survival age is 37.
Federation of American Societies for Experimental Biology
Cystic Fibrosis Current Events and Cystic Fibrosis News RSSResearchers describe how cells take out the trash to prevent disease
Garbage collectors are important for removing trash; without them waste accumulates and can quickly become a health hazard. Similarly, individual cells that make up such biological organisms as humans also have sophisticated methods for managing waste.
Children with cystic fibrosis not well covered by guidelines for vitamin D needs
Existing recommendations for treating vitamin D deficiency in children with cystic fibrosis (CF) are too low to cover the serious need, leaving most at high risk for bone loss and rickets, according to researchers at Johns Hopkins Children's Center.
New approach to treating cystic fibrosis lung infection shows promise
Researchers at the University of Calgary have found a new method of fighting severe lung infections in people with cystic fibrosis (CF). These findings are published in Proceedings of the National Academy of Science USA, this week.
Protein opens hope of treatment for cystic fibrosis patients
Scientists have finally identified a direct role for the missing protein that leaves cystic fibrosis patients open to attack from lung-damaging bacteria, the main reason most of them die before their 35th birthday, scientists heard today (Thursday 11 September 2008) at the Society for General Microbiology's Autumn meeting being held this week at Trinity College, Dublin.
Rattlesnake-type poisons used by superbug bacteria to beat our defenses
Colonies of hospital superbugs can make poisons similar to those found in rattlesnake venom to attack our bodies' natural defences, scientists heard today (Monday 8 September 2008) at the Society for General Microbiology's Autumn meeting being held this week at Trinity College, Dublin.
PTC124 shows activity in cystic fibrosis; Phase 2 proof-of-concept data published in Lancet
New phase 2 data published today in The Lancet show that the investigational oral drug PTC124 demonstrates activity in nonsense-mutation cystic fibrosis (CF). The data show that treatment with PTC124 results in statistically significant improvements in the chloride channel function of patients with nonsense-mutation CF. The study was conducted at the Hadassah Hebrew University Hospital in Jerusalem, Israel and sponsored by PTC Therapeutics (PTC).
Breaking the 'mucus barrier' with a new drug delivery system
Chemical engineers from Johns Hopkins University have broken the "mucus barrier," engineering the first drug-delivery particles capable of passing through human mucus - regarded by many as nearly impenetrable - and carrying medication that could treat a range of diseases. Those conditions include lung cancer, cervical cancer and cystic fibrosis, the research noted in a presentation scheduled for the 236th National Meeting of the American Chemical Society.
NIH scientists find a novel mechanism that controls the development of autoimmunity
Scientists at the National Institutes of Health (NIH) have found a mechanism in the immune systems of mice that can lead to the development of autoimmune disease when turned off.
Researchers discover cell's 'quality control' mechanism
Researchers in Japan and Canada have discovered a key component of the quality control mechanism that operates inside human cells - sometimes too well. The breakthrough has significant implications for the development of new treatments for cystic fibrosis (CF) and some other hereditary diseases, the researchers say. Their results were published July 25 in the journal Science.
Most generalists reluctant to provide primary care for young adults with chronic illness
The majority of general internists and pediatricians in the United States are not comfortable serving as primary care providers for young adults with complex chronic illnesses that originate during childhood, according to findings from a new national survey.
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