Research Shows Promise for Cystic Fibrosis and HIV Therapies

Innovative therapies against cystic fibrosis have shown promise for increasing the effectiveness of antibiotics in the treatment of chronic and acute bacterial respiratory infections in cystic fibrosis patients, and may also provide a model for potential HIV therapies in the future.

A number of compounds that block a key protein (exoenzymeS or ExoS) have been identified by Professor Igor Stagljar of the University of Toronto, with one—exosin—inhibiting infections in mammalian cells.

Past studies have shown it is possible to prevent or delay the onset of certain chronic or deadly infections in cystic fibrosis patients with early antibiotic treatment. But the current availability of antibiotics against Pseudomonas aeruginosa, a pathogen that can cause urinary tract infections, respiratory system infections, dermatitis, soft tissue infections, bacteremia, bone and joint infections, gastrointestinal infections and a variety of systemic infections, is limited and the pathogen shows signs of drug resistance.

In an article published in the journal PLoS Genetics, a team of investigators identified several drugs that block a Pseudomonas aeruginosa toxin called ExoS.

"These studies created a road map to the rational design of more potent, highly selective inhibitors against other similar toxins using a totally novel yeast-based approach," says lead author Stagljar. "This innovative approach is an important advance, not only for the value it may have in cystic fibrosis treatment, but also because this technique could be used to design novel therapies for any bacterial pathogen as well as the HIV virus."

Staglar’s next step is to test the action of their inhibitors in an animal model of cystic fibrosis, which if successful may provide a way for the treatment ofthis debilitating disease.
In the next phase of their research, Stagljar and his colleagues plan to test the action of their inhibitors in an animal model of cystic fibrosis. If successful, the therapeutics may provide an avenue for the treatment of this debilitating disease.

Source: University of Toronto


  1. quiact says

    Chromosome Alone 1: Cystic Fibrosis

    Cystic fibrosis (CF) is known as what is called an autosomal recessive genetic disorder, which means the disorder is present in such people because they lack an essential chromosome, which is what delivers DNA.
    With cystic fibrosis, each of your parents contributed to you what are known as CFTR protein genes that were mutated, meaning the genes are defective, which is why one acquires CF. The disease was first recognized during the 1930s. The name of the disease is derived from the frequent scarring of the pancreas associated with CF. CF has also been called Anderson’s Syndrome in the past.
    It is the most common hereditary disease in the United States. Also, the disease is almost entirely present in those who are ethnic white people and those of European decent. About 30,000 people in the U.S. have Cystic Fibrosis. Also, and for reasons unknown, men live longer than women with CF. This is disease is rare, yet devastating for one who may have it.
    This is a multi-system disease that can be deadly, and most with CF never lived past 5 years of age until the 1950s. Today, about 40 percent of cystic fibrosis patients are adults. With proper dietary supplements, and antibiotics to treat lung infections, the lifespan of CF patients continues to increase.
    Normally, CF is diagnosed when one is an infant- based on such things as low birth weight. Malnutrition is common due to the disease preventing the body from absorbing nutrients, and certain vitamins, such as vitamin D,E,A, or K. About 85 percent OF CF patients are deficient with such vitamins. In addition, genetic screening is now done on all neonates for up to 29 genetic disorders in most states in the U.S.
    Aside from the CF patient experiencing often pancreatic fibrosis, CF also causes lung infections and digestive problems as well. With cystic fibrosis, the glands in your exocrine system, glands that normally produce thin and slippery fluids for your internal organs instead produce thick mucus that essentially clogs your organs, such as your pancreas (mucovisidosis). This thick mucus also contributes to lung dysfunctions and infections as well.
    There are pharmaceuticals that have yet to be approved designed to treat cystic fibrosis patients, which appear to be promising for those affected by this disease, and improving their lifestyle, if not their lifespan..
    The CF patient may want to consider seeking out those doctors and hospitals who are specialists with cystic fibrosis for evaluative reasons and treatment reviews that will be best for them to address the complications of cystic fibrosis.
    Dan Abshear

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