Excellent Hair Fall Treatment
Search: Look for:   Last 1 Month   Last 6 Months   All time

Novel compounds for treating muscular dystrophy created

Washington , Thu, 23 Feb 2012 ANI
null

Washington, Feb 23 (ANI): Scripps Research Scientists have for the first time designed a series of small molecules that act against an RNA defect directly responsible for the most common form of adult-onset muscular dystrophy.

 

In two related studies, scientists from the Florida campus of The Scripps Research Institute found that these novel compounds significantly improve a number of biological defects associated with myotonic dystrophy type 1 in both cell culture and animal models.

 

"Our compounds attack the root cause of the disease and they improve defects in animal models," said Scripps Research Associate Professor Matthew Disney, PhD.

 

"This represents a significant advance in rational design of compounds targeting RNA. The work not only opens up potential therapies for this type of muscular dystrophy, but also paves the way for RNA-targeted therapeutics in general," Disney noted.

 

Myotonic dystrophy type 1 involves a type of RNA defect known as a "triplet repeat," a series of three nucleotides repeated more times than normal in an individual's genetic code.

 

In this case, the repetition of the cytosine-uracil-guanine (CUG) in RNA sequence leads to disease by binding to a particular protein, MBNL1, rendering it inactive. This results in a number of protein splicing abnormalities. Symptoms of this variable disease can include wasting of the muscles and other muscle problems, cataracts, heart defects, and hormone changes.

 

To find compounds that acted against the problematic RNA in the disease, Disney and his colleagues used information contained in an RNA motif-small molecule database that the group has been developing.

 

By querying the database against the secondary structure of the triplet repeat that causes myotonic dystrophy type 1, a lead compound targeting this RNA was quickly identified. The lead compounds were then custom-assembled to target the expanded repeat or further optimized using computational chemistry. In animal models, one of these compounds improved protein-splicing defects by more than 40 percent.

 

"There are limitless RNA targets involved in disease; the question is how to find small molecules that bind to them. We've answered that question by rationally designing these compounds that target this RNA. There's no reason that other bioactive small molecules targeting other RNAs couldn't be developed using a similar approach," Disney said.

 

The work has been published recently in online-before-print editions of Journal of the American Chemical Society and ACS Chemical Biology. (ANI)

 

null


null
LATEST IMAGES
King of Bhutan, the Bhutan Queen and Crown Prince meeting the PM Modi
PM Narendra Modi welcomes the King of Bhutan
PM Modi paying tributes at the portrait of Sardar Vallabhbhai Patel
People take part in the Run For Unity on the Rashtriya Ekta Diwas
PM Modi flagging off the Run For Unity on the Rashtriya Ekta Diwas
null
null
Post comments:
Your Name (*) :
Your Email :
Your Phone :
Your Comment (*):
  Reload Image
 
 

Comments:


   

OTHER TOP STORIES


Excellent Hair Fall Treatment
Careers | Privacy Policy | Feedback | About Us | Contact Us | | Latest News
Copyright © 2015 NEWS TRACK India All rights reserved.