HOME >> BIOLOGY >> NEWS
Cystic fibrosis splicing experiments offer hope for improved treatments

CHAPEL HILL -- Although not yet ready to try their new technique in patients, University of North Carolina at Chapel Hill scientists have successfully repaired a genetic problem that accounts for a form of cystic fibrosis commonly seen in patients of European Jewish descent.

Drs. Kenneth Friedman, research fellow in pathology and laboratory medicine, and Ryszard Kole, professor of pharmacology, at the UNC-CH School of Medicine developed the method to correct a mutation involving unnecessary information inside a gene. When that gene is imperfect, cystic fibrosis results.

The two conducted the research in collaboration with Drs. Lawrence Silverman and Michael Knowles at UNC-CH and Dr. Jonathan Cohn and technician Jolanta Kole at Duke University.

"This work builds on a strategy first explored by Dr. Kole in his studies of beta-thalassemia, a genetic disease often seen in Mediterranean and some Asian countries and involving defective hemoglobin molecules that lead to red blood cell destruction in bone marrow," Friedman said. "We applied his method to a cystic fibrosis gene mutation discovered here at UNC-CH in 1991 and given the somewhat arcane name 3849+10kbC>T."

That mutation results in a milder-than-most, but still debilitating, form of the illness and many years of chronic lung problems, he said. Cystic fibrosis, the most common lethal inherited disease among whites, is a complicated and highly variable condition resulting from errors in the gene controlling salt and water balance in the lungs and other tissues.

A report on their work appears in the Dec. 17 issue of the Journal of Biological Chemistry.

"We first genetically engineered healthy cells in laboratory culture to carry a version of this mutation, and, as expected, messenger RNA production in these cells was abnormal," Friedman said. "We then explored whether the effects of this mutation could be overcome by administration of pharmacological agents collectively known
'"/>

Contact: David Williamson
David_Williamson@unc.edu
919-962-8596
University of North Carolina at Chapel Hill
15-Dec-1999


Page: 1 2 3

Related biology news :

1. Cystic fibrosis gene linked to fatty acid defects
2. Cystic fibrosis gene therapy trial results encouraging
3. Cystic fibrosis gene mutations missing from some cases
4. Cystic fibrosis patients show too little salt in lungs, will need long-lasting treatments
5. Cystic fibrosis gene undetected in screening of infertile men
6. UI Researchers Find Potential Gene Therapy For Cystic Fibrosis
7. Cystic Fibrosis Conference Will Showcase Latest From Clinical Care To Basic Science
8. Lack Of Intestinal Bacterium Linked To Kidney Stones In Cystic Fibrosis Patients, University Of Florida Researchers Report
9. Debilitating Pancreatic Disorder Linked To Cystic Fibrosis Gene
10. Shellfish Toxin Study Provides Possible Clue To Cystic Fibrosis Therapy
11. Successful Specialist Care For Cystic Fibrosis Patients From Childhood To Adulthood

Post Your Comments:
(Date:3/26/2015)... The Granite Club, ... club, today announced it has implemented a state-of-the-art access ... for members and staff, while restricting access to authorized ... selected FST,s IMID Access system because it has the ... and staff, in addition to unparalleled security," said ...
(Date:3/24/2015)... 2015   NexID Biometrics LLC, whose spoof-mitigation ... today announced the beginning of shipments of version 2.0 ... The company, based in Potsdam, N.Y. ... Expo, which began here today at the Walter E. ... of its SDK boosts the accuracy rate range to ...
(Date:3/23/2015)... --  HOYOS Labs , a leading global authentication infrastructure ... Company will demonstrate multiple biometrics-based technologies under its identity ... on March 23 through 25, 2015, in ... IEEE Biometric Open Protocol Standard; free biometrics-based consumer app, ... system. BOPS was invented by HOYOS Labs and contributed ...
Breaking Biology News(10 mins):Granite Club First Private Club in North America to Implement FST Biometrics Secure Access Solution, as Part of Multi-million Dollar Expansion 2NexID Begins Shipments Of Version 2.0 Fake-Finger-Detection Solution 2HOYOS Labs to Showcase Host of Biometrics Technologies at Connect:ID in Washington, D.C. 2HOYOS Labs to Showcase Host of Biometrics Technologies at Connect:ID in Washington, D.C. 3
(Date:4/23/2015)... Six startups from Russia traveled ... as part of the U.S.-Russia Innovation Corridor (USRIC) ... biotechnology startups to participate in the program since ... the University of Maryland (UMD) signed a Memorandum ... universities in the biomedical industry in 2013. , ...
(Date:4/23/2015)... SAN FRANCISCO, Calif. , April 23, 2015  Veracyte, ... has entered into a definitive agreement to sell approximately ... placement. Under the agreement, the investors will purchase an ... purchase price of $8.15 per share, the closing stock ... led by new and existing investors, including Broadfin Capital, ...
(Date:4/23/2015)... 2015  Propanc Health Group Corporation (OTCBB: PPCH) ... the development of cancer treatments for patients with ... includes PRP, a patented formulation consisting of two ... as the enzyme amylase designed to synergistically enhance ... of anti-cancer agents working in combination with pro-enzymes, ...
(Date:4/22/2015)... -- Sangamo BioSciences, Inc. (NASDAQ: SGMO ) today reported ... For the first quarter ended March 31, 2015, ... or $0.08 per share, compared to a net loss ... same period in 2014. As of March 31, 2015, ... interest receivable of $226.1 million. Revenues for ...
Breaking Biology Technology:U.S.-Russian Innovation Corridor Program Connects Visiting Russian Startups to U.S. Experts 2U.S.-Russian Innovation Corridor Program Connects Visiting Russian Startups to U.S. Experts 3U.S.-Russian Innovation Corridor Program Connects Visiting Russian Startups to U.S. Experts 4Veracyte, Inc. Announces $40 Million Private Placement 2Veracyte, Inc. Announces $40 Million Private Placement 3Veracyte, Inc. Announces $40 Million Private Placement 4Early Stage BioTech offers Plenty of Risk; Propanc Health Group Analyst Brief Issued by BrokerBank Securities, Inc. 2Sangamo BioSciences Reports First Quarter 2015 Financial Results 2Sangamo BioSciences Reports First Quarter 2015 Financial Results 3Sangamo BioSciences Reports First Quarter 2015 Financial Results 4Sangamo BioSciences Reports First Quarter 2015 Financial Results 5Sangamo BioSciences Reports First Quarter 2015 Financial Results 6Sangamo BioSciences Reports First Quarter 2015 Financial Results 7Sangamo BioSciences Reports First Quarter 2015 Financial Results 8
Cached News: