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:3/11/2016)... -- http://www.apimages.com ) - --> http://www.apimages.com ) ... AP Images ( http://www.apimages.com ) - Germany ... the new refugee identity cards. DERMALOG will be unveiling this device, ... Hanover next week.   --> Germany ... produce the new refugee identity cards. DERMALOG will be unveiling this ...
(Date:3/10/2016)... --> --> ... and Access Management Market by Component (Provisioning, Directory Services, ... Organization Size, by Deployment, by Vertical, and by Region ... market is estimated to grow from USD 7.20 Billion ... a Compound Annual Growth Rate (CAGR) of 12.2% during ...
(Date:3/9/2016)... This BCC Research report provides an overview ... Sequencing (RNA Seq) market for the years 2015, 2016 ... reagents, data analysis, and services. Use this ... market such as RNA-Sequencing tools and reagents, RNA-Sequencing data ... each segment and forecast their market growth, future trends ...
Breaking Biology News(10 mins):
(Date:5/26/2016)... San Diego, CA (PRWEB) , ... May 26, ... ... assay development and manufacturing company, today announced several positive developments that position the ... As a result of the transaction, Craig F. Kinghorn has been appointed ...
(Date:5/25/2016)... , ... May 25, 2016 , ... ... a Thai delegation at BIO 2016 in San Francisco. Located at booth number ... be available to answer questions and discuss the Thai biotechnology and life sciences ...
(Date:5/25/2016)... Md. (PRWEB) , ... May 25, 2016 , ... ... request for information (RFI) issued by the Office of the National Coordinator for ... patient experience, and determines if clinically relevant data were available when and where ...
(Date:5/25/2016)... ... May 25, 2016 , ... Lady had been battling arthritis since the ... in her left knee. Lady’s owner Hannah sought the help of Dr Jeff Christiansen ... to repair her cruciate ligament and help with the pain of Lady’s arthritis. Dr ...
Breaking Biology Technology:
Cached News: