Navigation Links
Insights into a new therapy for a rare form of cystic fibrosis
Date:10/28/2012

Scientists at the Hospital for Sick Children in Toronto have established that a drug recently approved by the U.S. Food and Drug Administration to treat a rare form of cystic fibrosis works in an unconventional way. Their results reveal new possibilities for treating various forms of cystic fibrosis.

Cystic fibrosis is an inherited disease afflicting about 70,000 people around the world. Cystic fibrosis patients carry a defective gene that disables or destroys its protein product, which normally regulates the transport of ions across cell borders. When that transport is disrupted, the viscosity of the mucus coating certain organs becomes too thick. A characteristic feature of the disease is thick mucus buildup in the air passages, which causes difficulty breathing and recurring infections.

While the FDA approved the drug VX-770 (also known by the trade names Kalydeco and Ivacaftor) to ease breathing in people with cystic fibrosis caused by a particular mutation in the CFTR protein (the acronym is short for cystic fibrosis transmembrane conductance regulator), exactly how VX-770 worked in those patients was unknown.

Scientists have understood for some time that normal CFTR regulation requires modification of the protein and binding of a small, energy-providing molecule adenosine triphosphate, or ATP. But, in their recent Journal of Biological Chemistry "Paper of the Week," Christine Bear and colleagues report that the drug opens both normal and mutant CFTR channels without ATP. Their results indicate that the compound binds to a different site on CTFR than ATP. Significantly, this finding may be useful in developing therapies for cystic fibrosis caused by various CFTR mutations that, like the G551D mutation that was studied, impair ATP-mediated channel regulation.

Bear's group determined how VX-770 works after developing a new experimental system that may have potential for discovering drugs that target the basic defects caused by CFTR mutations, Bear says. The system is useful for identifying compounds that interact with rare mutations such as G551D as well as the major CFTR mutant F508del, she said.


'/>"/>
Contact: Angela Hopp
ahopp@asbmb.org
240-283-6614
American Society for Biochemistry and Molecular Biology
Source:Eurekalert

Related biology news :

1. New insights into cloud formation
2. Research on flavanols and procyanidins provides new insights into how these phytonutrients may positively impact human health
3. Battle of the sexes offers evolutionary insights
4. Study provides new insights into structure of heart muscle fibers
5. Study offers new insights into the effects of stress on pregnancy
6. Cell biology -- new insights into the life of microtubules
7. Yak genome provides new insights into high altitude adaptation
8. Zebrafish provide insights into causes and treatment of human diseases
9. Feces fossils yield new insights into ancient diets and thrifty genes
10. Piglets in mazes provide insights into human cognitive development
11. The ENCODE Project publishes new genomic insights in special issue of Genome Research
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:3/17/2016)... March 17, 2016 ABI Research, the ... the global biometrics market will reach more than ... increase from 2015. Consumer electronics, particularly smartphones, continue ... sensors anticipated to reach two billion shipments by ... Dimitrios Pavlakis , Research Analyst at ABI Research. ...
(Date:3/14/2016)... , Allemagne, March 14, 2016 ... http://www.apimages.com ) - --> - Renvoi : ... - --> --> ... solutions biométriques, fournit de nouveaux lecteurs d,empreintes digitales ... LF10 de DERMALOG sera utilisé pour produire des ...
(Date:3/10/2016)... --  Unisys Corporation (NYSE: UIS ) today announced ... testing its biometric identity solution at the Otay Mesa border ... identify certain non-U.S. citizens leaving the country. ... determine the efficiency and accuracy of using biometric technologies in ... until May 2016. --> the United States ...
Breaking Biology News(10 mins):
(Date:4/29/2016)... ... April 29, 2016 , ... Amendia, Inc., a ... procedures, today announced the completion of a significant transaction and partnership that positions ... customers and partners. Kohlberg & Company, L.L.C. (“Kohlberg”), a leading private equity ...
(Date:4/29/2016)... , April 29, 2016 Elekta ... latest update to its industry-leading treatment planning software, is ... Monaco version 5.11 provides significant ... attain calculation speeds up to four times faster than ... With the industry,s gold standard Monte Carlo ...
(Date:4/28/2016)... (PRWEB) , ... April 28, 2016 , ... ... made significant investments in recruiting top industry experts, and expanding its LATAM network ... provides industry-leading tools for clients to manage their clinical trial projects. , The ...
(Date:4/27/2016)... York, NY (PRWEB) , ... April 27, 2016 ... ... realizing it. Touch screen mobile devices with fingerprint recognition for secure access, ... libraries are only a few ways consumers are interacting with biometrics technology today. ...
Breaking Biology Technology: