Navigation Links
Discovery could increase efficacy of promising cystic fibrosis drug
Date:3/21/2013

A little more than a year after the FDA approved Kalydeco (Vx-770), the first drug of its kind to treat the underlying cause of cystic fibrosis, University of Missouri researchers believe they have found exactly how this drug works and how to improve its effectiveness in the future. Described in the current issue of the Proceedings of the National Academy of Sciences, MU researchers have redefined a key regulatory process in the defective protein responsible for cystic fibrosis that could change the way scientists approach the lethal genetic disease.

"They know the drug works, but they don't know how it works or where it works," said Tzyh-Chang Hwang, PhD, PNAS corresponding author and professor of medical pharmacology and physiology at the MU School of Medicine. "Our paper provides a theory for how Vx-770 works, and based on our understanding of how the CFTR channel works, we have identified a novel strategy for future explorations to complement and enhance the performance of the existing drug."

Cystic fibrosis is the second most common life-shortening inherited disorder occurring in childhood in the United States, after sickle cell anemia. Approximately 30,000 Americans have cystic fibrosis, and there are an estimated 1,000 new cases diagnosed each year. Cystic fibrosis patients are born with a genetic defect that causes a malfunction of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, a chloride channel in the cell membrane that plays a critical role in maintaining water and salt balance across many body tissues, such as sweat glands, tissues that line the lungs, liver, pancreas and reproductive organs.

"The chloride channel is like a pipe that allows ions to travel through at a very fast pace," Hwang said. "In cystic fibrosis patients the channel is dysfunctional and activity is diminished. So what is the mechanism that controls the opening and closing of the channel? That is the fundamental disco
'/>"/>

Contact: Michael Muin
muinm@health.missouri.edu
573-884-7541
University of Missouri School of Medicine
Source:Eurekalert  

Page: 1 2 3 4

Related biology news :

1. Discovery of a molecule that initiates maturation of mammalian eggs can lead to more IVF pregnancies
2. Annual Drug Discovery Conferences Being Held in Boston MA, Spring 2012
3. Discovery provides blueprint for new drugs that can inhibit hepatitis C virus
4. Unexpected discovery reveals a new mechanism for how the cerebellum extracts signal from noise
5. Discovery offers insight into treating viral stomach flu
6. Breast cancer risk gene discovery fast tracked by new technology
7. Tales from the crypt lead researchers to cancer discovery
8. New discovery may lead to effective prevention and treatment of graft-versus-host dsease
9. Stomata development in plants unraveled -- a valuable discovery for environmental research
10. Discovery reveals chromosomes organize into yarns
11. Bacteria discovery could lead to antibiotics alternatives
Post Your Comments:
*Name:
*Comment:
*Email:
Related Image:
Discovery could increase efficacy of promising cystic fibrosis drug
(Date:12/24/2014)... in December 2014, the 1U™ app has helped ... remember their usernames and passwords through replacing the antiquated system ... people who have struggled to remember usernames and passwords, ... on redefining identity, announced today that it is offering the ...
(Date:12/22/2014)... 22, 2014  The 2014 Holiday Season may be the ... Intelligence reports that the long anticipated floodgates for consumer ... demand for smart phones, tablets, and wearable mobile devices ... 2.5 billion users with nearly 4.8 billion biometric devices ...
(Date:12/19/2014)... 2014  23andMe, Inc., the leading personal genetics company, today announced ... genetic ancestry of individuals from across the United ... than four hundred years ago, the United States ... from different continents. This study illuminates how American history and ...
Breaking Biology News(10 mins):1U Offers Best Solution to the Username / Password Dilemma: For FREE! 21U Offers Best Solution to the Username / Password Dilemma: For FREE! 3First Season of Holiday Shopping with Mobile Biometric Payments Wraps Up With a Present for Biometrics Industry: A Rosy Forecast for More Than 2 Billion Users of 4.8 Billion Mobile Biometric Devices by 2020 223andMe Study Sketches Genetic Portrait of the United States 223andMe Study Sketches Genetic Portrait of the United States 323andMe Study Sketches Genetic Portrait of the United States 4
... share a biological adaptation that enables them to thrive in ... pass on the trait appears to be linked to different ... Reserve University scientist and colleagues shows. The adaptation is ... level of hemoglobin, the oxygen-carrying protein in red blood cells. ...
... Notre Dame stream ecologist Gary Lamberti. If you,re catching ... strong salmon run, the stream fish brook trout, brown ... by the salmon. Research by Lamberti, professor and chair ... they travel upstream to spawn and die, carry industrial pollutants ...
... ESA joined international delegates in Doha, Qatar, to discuss how ... climate change and how this information is useful to ... talks and negotiations, the UN Climate Change Conference has entered ... Ministers of Environment. ,During the first week of the ,COP ...
Cached Biology News:Different genes behind same adaptation to thin air 2Notre Dame research reveals migrating Great Lakes salmon carry contaminants upstream 2Notre Dame research reveals migrating Great Lakes salmon carry contaminants upstream 3Climate - the hot topic in Doha 2
(Date:1/22/2015)... 22, 2015 Diagenode, Inc., a ... the Bioruptor® and complete solutions for epigenetics research, ... immunoprecipitation, alleviating the need for manual processing. The ... for ChIP of histones or transcription factors and ...
(Date:1/22/2015)... , Jan. 22, 2015  Derma Sciences, Inc. (Nasdaq: ... focused on advanced wound care, announces that AMNIOEXCEL® ... have been added to the Premier, Inc. Regenerative ... opportunity for the AMNIOEXCEL® and AMNIOMATRIX® product lines, ...
(Date:12/24/2014)... Dec. 24, 2014   BioLife Solutions , Inc. (NASDAQ: ... proprietary clinical grade hypothermic storage and cryopreservation ... for cells and tissues  ("BioLife" or the "Company"), today announced ... on May 4, 2015 (the "Annual Meeting"). Because ...
(Date:12/24/2014)... Bethesda, MD (PRWEB) December 23, 2014 ... law H.R. 83, the Omnibus and Continuing Resolution Appropriations ... hydrocephalus a condition eligible to receive funding through the ... Department of Defense (DoD). The Hydrocephalus Association (HA), working ...
Breaking Biology Technology:Diagenode Launches Unique Semi-Automated Solution for Chromatin Immunoprecipitation 2Derma Sciences Expands Access of its AMNIOTIC TISSUE Product Line with New Premier, Inc. Agreement 2Derma Sciences Expands Access of its AMNIOTIC TISSUE Product Line with New Premier, Inc. Agreement 3Derma Sciences Expands Access of its AMNIOTIC TISSUE Product Line with New Premier, Inc. Agreement 4Derma Sciences Expands Access of its AMNIOTIC TISSUE Product Line with New Premier, Inc. Agreement 5BioLife Solutions Sets Date for Annual Meeting of Stockholders 2Rare Brain Condition Now Eligible for Department of Defense Research Dollars 2
... CLEVELAND, March 19, 2012 The nation,s first ... 21-22 in Cleveland.  MBEC offers a unique platform ... leaders to engage, network, collaborate and exchange ideas. ... Minorities represent less than 5 percent(1) of ...
... that the product contains L(+)-lactic acid, one of two forms ... two"isomers", L(+) and D(-), have different "optical activities", turning the ... Of course the capacity to alter the polarization, intensity or ... Light interacts in one way or another with all matter. ...
... ST. LOUIS, March 15, 2012 Sigma-Aldrich® Corporation (NASDAQ: ... its new facility in Wuxi, China, that will provide ... products to support the Company,s research and industrial customers ... China has invested heavily in industrial growth, and now ...
Cached Biology Technology:Minority Biomedical Entrepreneurship Conference 2Minority Biomedical Entrepreneurship Conference 3Designer lights from the physics lab 2Sigma-Aldrich Opens New Wuxi Packaging and Quality Control Facilities to Enhance Capabilities Serving China and Asia-Pacific Region 2Sigma-Aldrich Opens New Wuxi Packaging and Quality Control Facilities to Enhance Capabilities Serving China and Asia-Pacific Region 3
... SB3, Fully adjustable mixing angle, Variable speed, ... hold a number of different sized tubes, ... aerating cultures, keeping biological samples in suspension ... can be used in incubators up to ...
... 2 real-time PCR detection system uses a ... (470-505 nm) and two photomultiplier tubes for ... Opticon 2 system is built on an ... thermal gradient feature. This item includes the ...
... Multicolor capability allows detection of SYBR ... channel, and a range of fluorophores in ... VIC, and TAMRA for a multitude ... discrimination. DNA Engine thermal cycler offers precision ...
Fingerprinting II dimensioning techniques is an optional module that adds principal component analysis, multidimensional scaling, discriminant analysis and MANOVA, and self-organizing maps to the Fin...
Biology Products: