Researchers Uncover Mechanism for Potential Friedreich's Ataxia Drug

by Rajashri on  September 29, 2009 at 9:01 PM Drug News
  •   Print
  •   Share
  •   Comments
  •  Text 
The enzyme target of a drug group that stops the progression of the devastating disease Friedreich's ataxia in mice has been pinpointed by a Scripps research team.

The findings, developed in collaboration with scientists from Repligen Corporation, help advance this treatment approach one step closer toward human clinical trials, which will be a welcome event for disease sufferers who currently have few treatment options.

The work, reported as the cover article of the September 25, 2009 issue of the journal Chemistry & Biology, could also lead to treatments for related conditions such as Huntington's disease and the spinocerebellar ataxias.

"It will be very rewarding if our work actually leads to a therapy for Friedreich's," says Joel Gottesfeld, a professor in the Department of Molecular Biology and leader of the Scripps Research team that discovered the potential treatment. "This is a horrible disease."

Friedreich's ataxia, which afflicts about one of every 20,000 to 50,000 people in the United States, is caused by inadequate production of the protein frataxin, which leads to degeneration of nerve tissue and an array of associated complications including heart disease and scoliosis. In most cases, sufferers are ultimately confined to a wheelchair and many die as young adults.

Researchers have tied this low frataxin production to a large repetition of a specific triplet DNA pattern in the frataxin gene. Though many questions remain open, it appears that the unusual DNA structure resulting from these repetitions somehow attracts enzymes known as histone deacetylases (HDACs). These enzymes alter the packaging of the DNA in chromosomes in a way that inactivates the expression of the frataxin gene, though it remains intact.

Page 1 Page 1 | 2  Next
 Email Email   RSS Feeds RSS Feeds   Print this page Print   Save this page Save   Link Link   Syndicate Syndicate   Comments Comments   Bookmark and Share
 
Comment & Contribute
Comments should be on the topic and should not be abusive. Comments are normally moderated and are reviewed after they are posted.
* Your comment can be maximum of 2500 characters

Notify me when reply is posted
I agree to the terms and conditions
  
If you have a question about health related issues, you can now post it in our Ask An Expert section on our community website Medwonders.com and get answers from our panel of experts.
X

Medwonders Health Network

  • Health News Index
A B C D E F G H I J K L M N O P Q R S T U V W X Y Z
News Archive
Date :
Category :
Keyword :
  • News Quick Links
News Central Health Watch
Latest Health News Health In Focus
News Category (500+) Breaking Health News
Popular News Celebrating Life
Health News and Press Release Medindia - Exclusive
News Photo Gallery India Special
News Video Gallery Lifestyle and Wellness
News From Other Resources
Ataxia
Complete Medindia Resources
News Categories:  
Kidney Health Center

Drug Related News

» Schizophrenia Drug Differentiates Cancer Stem Cells into Less Threatening Cells » Docs Who Seldom Meet Drug Reps Continue to Prescribe Drugs With FDA “Black Box” Warnings
» Revision in Quality Standards for Heparin » Acne Drug Users At High Risk of Developing Eye Infections
» Patients Who Take Antidepressants When in ICU More Likely to Die » New Tablet Coating Product by Ideal Cures, In Geneva
» Prednisolone Benefits Patients With Bell's Palsy » 'Orphan' Sleep Drug may Fight Cancer
Read More >>