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 def
'/>"/>

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

Page: 1 2

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:11/4/2014)... amount of death at the right time might actually ... research that could help in understanding animal populations, pest ... a paper in the journal Trends in Ecology ... colleagues conclude that the kind of positive population effect ... or mortality, depends on the size and developmental stage ...
(Date:11/4/2014)... Think about the way our bodies are assembled during ... that they are supposed to become a nerve or ... the correct place and alignment? Researchers at the University ... In a new study, UM researchers describe the signaling ... neighbors, at the head-trunk region. Their discovery may have ...
(Date:11/4/2014)...   3D scanner developer targets ... Fuel3D , a developer of 3D scanning solutions, today announced ... (£4 million). This funding builds on the company,s initial $2.6 ... the way for the commercial launch of the company,s consumer ... led by Chimera Partners and will be used to ramp-up ...
Breaking Biology News(10 mins):When less is more: Death in moderation boosts population density in nature 2The inside story: How the brain and skull stay together 2Fuel3D Secures $6.4 Million in Expansion Funding 2Fuel3D Secures $6.4 Million in Expansion Funding 3
... NANO -- World,s smallest antenna . . . ... metal or dipole antenna, electronic devices of tomorrow could incorporate ... possible by a design that allows an electrically charged nano-mechanical ... be the days when we need to match the antenna ...
... the day a machine can draw your blood, screen it ... and pop out a drug tailor-made for your DNA. ... point irregularities which have accumulated over time that can lead ... National Institutes of Health has tapped Viterbi professor Andrea Armani ...
... Finally, a gazelle comes into view. The cheetah plunges forward, ... the hapless gazelle by the jugular in less than a ... A blue whale surfaces, blasting water high from its ... with air. Then it descends again to look for krill, ...
Cached Biology News:Story tips from the US Department of Energy's Oak Ridge National Laboratory October 2010 2Story tips from the US Department of Energy's Oak Ridge National Laboratory October 2010 3Fighting cancers by tagging their triggers 2Slicing proteins with Occam's Razor 2Slicing proteins with Occam's Razor 3Slicing proteins with Occam's Razor 4
(Date:11/21/2014)... 20, 2014 Polaris Group announced today ... its Phase 1 trial of ADI-PEG 20 in ... of malignant pleural mesothelioma (MPM) and non-squamous non-small ... trials on ADI-PEG 20, both as monotherapy and ... of several other indications, including breast cancer, melanomas, ...
(Date:11/21/2014)... SAN DIEGO , Nov. 20, 2014  Cytori Therapeutics ... Marc Hedrick , M.D. will present live at VirtualInvestorConferences.com ... 4, 2014 TIME:    11:15 am PT / 2:15 ... this URL into your browser,s address bar: http://bit.ly/1B2KGaL ... time and receive event updates. This will ...
(Date:11/18/2014)... SAN BRUNO, Calif. , Nov. 18, 2014 ... is a platinum sponsor of the 10th Annual World Stem ... and regenerative medicine stakeholders. The 2014 World Stem Cell Summit ... San Antonio, Texas . ... the discovery and development of lifesaving cures and therapies, convening ...
(Date:11/18/2014)... 17, 2014 Cirrascale Corporation ... blade-based and rackmount computing infrastructure, today announced it ... accelerator throughout its GPU-enabled blade server and high-performance ... company’s latest proprietary 80-lane Gen3 PCIe switch-enabled risers, ... Tesla K80 dual-GPU accelerator cards in a single ...
Breaking Biology Technology:Polaris Group Announces Treatment of First Patient in Phase 1 Study of ADI-PEG 20 plus Pemetrexed and Cisplatin in Malignant Pleural Mesothelioma And Non-Squamous Non-Small Cell Lung Carcinoma 2Cytori Therapeutics to Provide Corporate Update and Investor Presentation on December 4 through a Live, Interactive Webcast 2Cytori Therapeutics to Provide Corporate Update and Investor Presentation on December 4 through a Live, Interactive Webcast 3Cytori Therapeutics to Provide Corporate Update and Investor Presentation on December 4 through a Live, Interactive Webcast 4Cord Blood Registry Sponsors 2014 World Stem Cell Summit 2Cord Blood Registry Sponsors 2014 World Stem Cell Summit 3Cirrascale® Expands Multi-GPU Peering Solutions with Support for New NVIDIA Tesla K80 Dual-GPU Accelerators 2Cirrascale® Expands Multi-GPU Peering Solutions with Support for New NVIDIA Tesla K80 Dual-GPU Accelerators 3
... Stemedica ... of clinical grade biological products as licensed by the State of California Food and Drug ... latest scientific discoveries at the 2009 World Stem Cell Summit. , ... San Diego, CA (PRWEB) Aug 27, 2009 -- ...
... , The statement below is from Pete Nicholas, Chairman ... employees and its Board of Directors. , , ... remarkably broad spectrum of issues. Foremost among them was his long and ... , He was a tireless advocate for patients, rights and for ...
... , CHICAGO, Aug. 26 Ajinomoto Food ... adding food product development capabilities. The Application Center North America ... , The ACNA mission is to develop and ... technology applications will be the initial focus of the Center: ...
Cached Biology Technology:Stemedica Selected By World Stem Cell Summit To Present Scientific Discoveries 2Stemedica Selected By World Stem Cell Summit To Present Scientific Discoveries 3Stemedica Selected By World Stem Cell Summit To Present Scientific Discoveries 4Boston Scientific Statement on the Passing of Senator Edward Kennedy 2
... 48° Fixed Angle Polypropylene Rotor ... Series, IEC Centra CL3/CL3R** and Centra-MP4/MP4R*. ... quiet operation. /MP4, ,EXCLUSIVE polypropylene rotor ... can be repeatedly autoclaved. Polypropylene ...
The 891 45° Fixed Angle Rotor is designed to provide aerosol containment and can be removed from a centrifuge (seal entact) and opened under an environmental hood....
... The Xcise is a powerful technology ... throughput proteome analysis. High quality sample ... untrained user. , The Xcise is a ... for high throughput proteome analysis. It combines ...
Extra Large bottle for hybridization 300 x 70mm...
Biology Products: