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:2/24/2017)... ... , ... Only two months after the official release of The Private Collection ... XO Private has initiated a second print-run of its lavish luxury travel coffee table ... when open, weighs in at more than six kilos, retails at EUR 1,000 per ...
(Date:2/24/2017)... (PRWEB) , ... February 24, 2017 , ... In the ... to be a top priority because it’s not if you will be attacked, but ... especially when it comes to digital health care. , Improvements in auditing and monitoring ...
(Date:2/24/2017)... ... ... The California State University Institute for Palliative Care is pleased ... palliative care education and research. The Symposium, “Innovate. Investigate. Educate: Advancing Academic Palliative ... Sept. 28 and 29, 2017, on the campus of California State University San Marcos. ...
(Date:2/24/2017)... ... February 24, 2017 , ... Rosica Communications, a national ... , media relations, social media, content marketing and SEO, is now offering direct, ... an intuitive marketing automation platform. , Rosica will now offer the platform to ...
(Date:2/23/2017)... , ... February 23, 2017 , ... The 89th Academy ... winner of the 2016 National Education Policy Center Bunkum Award. We invite you to ... 2016. , This year’s Bunkum winner is the Center for American Progress (CAP), for ...
Breaking Medicine News(10 mins):
(Date:2/24/2017)... , Feb. 24, 2017 Juan ... & Associates PC , a boutique securities firm headquartered ... York City , announces that a class action ... Massachusetts  against Inotek Pharmaceuticals Corporation (NASDAQ: ... the Company,s securities between July 23, 2015 and December 30, ...
(Date:2/24/2017)... , Feb. 23, 2017  Directors from Pharma To Market Pty ... have joined forces, resulting in the founding of Pharma To Market ... Pharma To Market are pleased to announce their expansion into ... Singapore . The company are delighted to appoint ... of the Singapore based entity. Joelle brings ...
(Date:2/24/2017)... , Feb. 24, 2017  Xynomic Pharmaceuticals, Inc., ... announced that it has acquired exclusive worldwide rights ... best-in-class innovative HDAC inhibitor targeting hematological and solid ... 14 Phase 1 and 2 clinical trials of ... have already been completed, demonstrating that ...
Breaking Medicine Technology: