Navigation Links
Scripps Florida scientists create new approach to destroy disease-associated RNAs in cells
Date:12/20/2012

JUPITER, FL, December 20, 2012 Scientists from the Florida campus of The Scripps Research Institute (TSRI) have developed a new approach to alter the function of RNA in living cells by designing molecules that recognize and disable RNA targets. As a proof of principle, in the new study the team designed a molecule that disabled the RNA causing myotonic dystrophy.

The study, published online ahead of print on December 20, 2012 by the journal Angewandte Chemie, reports the creation of small molecules that recognize disease-associated RNAs, targeting them for destruction. Since small molecules are cell-permeable, the approach could have benefits over traditional methods of targeting RNAs for degradation, such as antisense or RNA interference (RNAi).

"We're excited about these results," said Matthew Disney, an associate professor at TSRI who pioneered the research. "This approach may allow for the inactivation of many cellular RNAs by small molecules and potentially lead the way to a whole range of novel therapeutics."

It's well known that gene expression can be controlled by triggering the degradation of messenger RNAthe blueprint for the production of proteins. This is accomplished through the recruitment of compounds that cleave or split the molecule. While several compounds can induce RNA cleavage in vitro, this has not been accomplished efficiently in living cellsuntil now.

In the new study, Disney and Research Associate Lirui Guan attached a rationally designed small molecule that targets the RNA that causes myotonic dystrophy type 1 with a molecule that produces hydroxyl radicals. Upon the small molecule's recognition of the target, a hydroxyl radical was released that cleaved the disease-associated RNA, alleviating the disease-associated defects. Disney noted that, despite the compound's producing a highly reactive species, the compounds are non-toxic at relatively effective doses.

The team accomplished this feat through what Disney calls a bottom-up approach to targeting RNA.

"We first identified the preferred RNA structural elements or motifs that bind to small molecules," he said. "Then we looked at these elements in RNAs that cause disease and designed a binding molecule with increased affinity and specificity for those elements."

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, resulting 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.

The applications for this new approach could include cancer treatment in conjunction with other therapies, Disney said. The approach could also be used to create chemical probes of RNA function or to develop tools to probe RNA structureprovided, of course, that the RNA-binding preferences of the small molecules involved were well defined.


'/>"/>

Contact: Eric Sauter
esauter@scripps.edu
267-337-3859
Scripps Research Institute
Source:Eurekalert  

Related medicine news :

1. Scripps Florida scientists develop new compound that reverses fatty liver disease
2. Scripps Florida scientist awarded $2.5 million to study inner workings of memory formation
3. Scripps Florida scientists uncover a novel cooperative effort to stop cancer spread
4. Scripps Research Institute study points to potential new therapies for cancer and other diseases
5. 2 Scripps Research Institute scientists honored by American Chemical Society
6. Scripps studies show community-based diabetes programs are key to lowered costs and improved care
7. Scripps Research Institute receives $20 million to shed light on HIV drug resistance
8. Scripps Research Institute scientists show protein linked to hunger also implicated in alcoholism
9. Scripps Research scientists devise powerful new method for finding therapeutic antibodies
10. Clinical trials start for stroke drug developed by Scripps Research, USC, and ZZ Biotech
11. Scripps Florida scientists awarded nearly $1.5 million to develop new approaches to treat cancer
Post Your Comments:
*Name:
*Comment:
*Email:
Related Image:
Scripps Florida scientists create new approach to destroy disease-associated RNAs in cells
(Date:10/13/2017)... ... ... Yisrayl Hawkins, Pastor and Overseer at The House of Yahweh, has released ... understood books in the Holy Scriptures, Revelation. The Book of Revelation paints a picture ... Many have tossed it off as mere rubbish, but Yisrayl Hawkins says that is ...
(Date:10/12/2017)... ... October 12, 2017 , ... First Healthcare Compliance (FHC), an ... showcase a range of technology and learning solutions at the 68th Annual American ... to be held October 14–18, 2017 at the Mandalay Bay Resort in Las ...
(Date:10/12/2017)... ... , ... The American College of Medical Informatics (ACMI) will present the 2017 ... Session of AMIA’s Annual Symposium in Washington, D.C. AMIA’s Annual Symposium is ... pioneer in the field of medical informatics, this prestigious award is presented to an ...
(Date:10/12/2017)... ... October 12, 2017 , ... Leading pediatric oncology experts ... Washington, D.C., for the 49th Congress of the International Society of Paediatric Oncology ... of the Center for Cancer and Blood Disorders at Children’s National, and ...
(Date:10/12/2017)... ... October 12, 2017 , ... Vohra Chief ... advancements to physician colleagues, skilled nursing facility medical directors and other clinicians at ... of Wound Care." , "At many of these conferences we get to educate ...
Breaking Medicine News(10 mins):
(Date:10/2/2017)... -- The Rebound mobile app is poised to become a ... of prescription drug addiction. The app empowers users to develop ... down their dosage in a safe, controlled manner while maximizing ... first 100,000 people to sign up will enjoy 3 months ... ...
(Date:9/27/2017)... YORK , Sept. 27, 2017  DarioHealth Corp. (NASDAQ: DRIO), a ... today announced that its MyDario product is expected to appear on The ... when The Dr. Oz Show airs in your area: http://www.doctoroz.com/page/where-watch-dr-oz-show ... The nine-time Emmy award-winning, The Dr. Oz ... The ...
(Date:9/22/2017)... , Sept. 22, 2017  As the latest ... Senators Bill Cassidy (R-LA) and Lindsey ... notes that the medical device industry is in an ... device tax, the 2.3% excise tax on medical device ... they also want covered patients, increased visits and hospital ...
Breaking Medicine Technology: