Scripps Florida Scientists Create New Approach to Destroy Disease-Associated RNAs in Cells As Proof of Principle, Team Creates Molecule that Corrects Myotonic Dystrophy in Living Cells
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 RNA the 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 cells until 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 structure provided, of course, that the RNA-binding preferences of the small molecules involved were well defined.
The study, Small Molecule-Mediated Cleavage of RNA in Living Cells, was supported by the National Institutes of Health (Grant number: R01-GM079235) and TSRI. For more information on the study, see http://onlinelibrary.wiley.com/doi/10.1002/anie.201206888/abstract.
Most recent headlines
02/01/2026
Share Share by:
Copy link
Facebook
X
Whatsapp
Pinterest
Flipboard...
02/01/2026
Sequins, chat shows, live sporting action, ground-breaking docuseries and brand-new Irish drama to kick off 2026
New Year, New Content Coming Soon across RT
...
01/01/2026
Latin Grammy Cultural Foundation Announces 2026 Noel Schajris Scholarship The scholarship will cover tuition and housing for one Berklee College of Music stud...
01/01/2026
New year, new games, all with RTX 5080-powered cloud energy. GeForce NOW is kicking off 2026 by looking back at an unforgettable year of wins and wildly high fr...
31/12/2025
Back to All News
NFL Christmas Gameday on Netflix Scores Again With the Lions-V...
30/12/2025
As the College Football Playoff Enters the Quarterfinals, ESPN Blows Out Its Meg...
30/12/2025
SVG's Best of 2025: Original ArticlesTake a look back at all our coverage of big-time productions, game-changing technologies, and state-of-the-art new faci...
30/12/2025
MELBOURNE, Fla., Dec. 30, 2025 - L3Harris Technologies (NYSE: LHX) will release its fourth quarter 2025 financial results before the market opens on Thursday, J...
30/12/2025
Share Share by:
Copy link
Facebook
X
Whatsapp
Pinterest
Flipboard...
30/12/2025
Your live countdown to 2026 with Inhaler, David Gray, Lyra, Garron Noone, Sharon...
30/12/2025
It marked the first civilian operational authorization for a HAPS flight in Europe, led by Space42's subsidiary, Mira Aerospace
The flight demonstrated HAP...
29/12/2025
San Francisco 49ers Strike Gold With Halftime Laser SpectacularStunning display caps $200 million renovation of Levi's Stadium techBy Dan Daley, Audio Edito...
29/12/2025
The Cup's Around the Corner: An Inside Look at Broadcast Preparations for th...
29/12/2025
SVG's Best of 2025: Longform VideoWatch the standout keynote conversations, deep dives, and panel discussions from the year for free on SVG PLAY!By Brandon ...
29/12/2025
From crisper Lossless audio and immersive music videos in beta to new Audiobooks+ plans, custom transitions between tracks, and in-app Messages, we keep levelin...
29/12/2025
Share Share by:
Copy link
Facebook
X
Whatsapp
Pinterest
Flipboard...
27/12/2025
Share Share by:
Copy link
Facebook
X
Whatsapp
Pinterest
Flipboard...
26/12/2025
Share Share by:
Copy link
Facebook
X
Whatsapp
Pinterest
Flipboard...
26/12/2025
Share Share by:
Copy link
Facebook
X
Whatsapp
Pinterest
Flipboard...
25/12/2025
Holiday lights are twinkling, hot cocoa's on the stove and gamers are settling in for a well-earned break.
Whether staying in or heading on a winter getawa...
24/12/2025
What is AI good for? Posted by MTI Film on December 24, 2025
What is AI good for?
What is AI good for?
It's been three years since ChatGPT first cap...
24/12/2025
Share Share by:
Copy link
Facebook
X
Whatsapp
Pinterest
Flipboard...
24/12/2025
Share Share by:
Copy link
Facebook
X
Whatsapp
Pinterest
Flipboard...
24/12/2025
Share Share by:
Copy link
Facebook
X
Whatsapp
Pinterest
Flipboard...
24/12/2025
Share Share by:
Copy link
Facebook
X
Whatsapp
Pinterest
Flipboard...
24/12/2025
Share Share by:
Copy link
Facebook
X
Whatsapp
Pinterest
Flipboard...
24/12/2025
Back to All News
The Boyfriend' Season 2 Unveils Heartwarming Trailer, Key...
24/12/2025
Back to All News
Love, Fights, and Everything in Between: Badly in Love' Returns for Season 2
Entertainment
24 December 2025
GlobalJapan
Link copied t...
24/12/2025
Scripps Research study links sleep variability with sleep apnea and hypertension How consumers' digital activity trackers could enable personalized health s...
23/12/2025
How guilas Cibae as Dominican Winter League Games Are Locally Produced for Glob...
23/12/2025
BitFire's Jim Akimchuk on Supplying Scalability and Customization in the Clo...
23/12/2025
CAMB.AI Enables European Athletics to Offer Multi-Language SupportPlan is to eventually offer translation into all languages spoken in EuropeBy Ken Kerschbaumer...
23/12/2025
Analysis: As sports media values trend negative, scarcity and quality are king By Callum McCarthy, Editor-at-Large
Monday, December 22, 2025 - 14:08
Print ...
23/12/2025
ESPN, Disney, and NBA Return to the Animated Altcast Fray With Second Edition of...
23/12/2025
End the Year on a High Note and Donate to the Sports Broadcasting Fund Today!By Ken Kerschbaumer, Editorial Director
Tuesday, December 23, 2025 - 12:25 pm
P...
23/12/2025
The year is winding down, the weather outside is frightful, and it's the perfect time to escape into a story that warms the heart. For listeners looking for...
23/12/2025
A Zeus motor is hot fire tested at L3Harris' Camden, Arkansas, solid rocket ...
23/12/2025
Share Share by:
Copy link
Facebook
X
Whatsapp
Pinterest
Flipboard...
23/12/2025
Share Share by:
Copy link
Facebook
X
Whatsapp
Pinterest
Flipboard...
23/12/2025
Lightware will exhibit several major product innovations at ISE 2026, including the new USB-C BOOSTER-V1, Google Meet. integration for various Taurus UCX models...
23/12/2025
Share Share by:
Copy link
Facebook
X
Whatsapp
Pinterest
Flipboard...
23/12/2025
Share Share by:
Copy link
Facebook
X
Whatsapp
Pinterest
Flipboard...
23/12/2025
Share Share by:
Copy link
Facebook
X
Whatsapp
Pinterest
Flipboard...
23/12/2025
Share Share by:
Copy link
Facebook
X
Whatsapp
Pinterest
Flipboard...
23/12/2025
Share Share by:
Copy link
Facebook
X
Whatsapp
Pinterest
Flipboard...
23/12/2025
Taking the Stage at Carnegie Hall-On a Global Scale Boston Conservatory Orchestra students reflect on their epic concert marking the 80th session of the UN Gene...
23/12/2025
Back to All News
Netflix's The Great Flood and Culinary Class Wars 2 Top Gl...
23/12/2025
Back to All News
Stranger Things By the Numbers: How the Global Phenomenon Shap...
23/12/2025
Experience the power of WO Automation for Radio's newest service, the System Effectiveness Review. Designed to help you achieve more, a System Effectiveness...
Scripps Florida Scientists Create New Approach to Destroy Disease-Associated RNAs in Cells As Proof of Principle, Team Creates Molecule that Corrects Myotonic Dystrophy in Living Cells
