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:
*Name:
*Comment:
*Email:


(Date:4/5/2017)... -- Today HYPR Corp. , leading innovator in ... the HYPR platform is officially FIDO® Certified . ... that empowers biometric authentication across Fortune 500 enterprises and ... 15 million users across the financial services industry, however ... suites and physical access represent a growing portion of ...
(Date:3/30/2017)... 30, 2017 The research team of The ... (3D) fingerprint identification by adopting ground breaking 3D fingerprint minutiae recovery ... of speed and accuracy for use in identification, crime investigation, immigration ... ... A research team ...
(Date:3/24/2017)... , March 24, 2017 The Controller General ... Controller Mr. Abdulla Algeen have received the prestigious international IAIR ... Continue Reading ... ... picture) and Deputy Controller Abdulla Algeen (small picture on the right) have ...
Breaking Biology News(10 mins):
(Date:8/16/2017)... ... 2017 , ... We are proud to announce the successful ... Dilworth, MN site. The inspection took place Monday, July 31st through Friday, August ... a routine Bioresearch Monitoring Program (BIMO) with the USFDA wherein multiple Dermatology Vasoconstriction ...
(Date:8/15/2017)... ... August 15, 2017 , ... Kapstone Medical is proud ... successes helping medical technology companies and inventors develop and safeguard their latest innovations. ... engineering firm with a portfolio of clients in the United States and around the ...
(Date:8/15/2017)... ... August 15, 2017 , ... Kenall, ... modular downlights designed to stay tightly sealed and perform efficiently for years. The ... wet location listings just aren't enough, such as: hospitals; behavioral health facilities; cleanrooms; ...
(Date:8/14/2017)... Ca (PRWEB) , ... August 14, 2017 , ... ... poorly characterized and performing antibodies. Key researchers in the antibody community have recently ... characterization and consistency for antibodies in the laboratory. , The team ...
Breaking Biology Technology:
Cached News: