PRESS-NEWS.org - Press Release Distribution
PRESS RELEASES DISTRIBUTION

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

Scripps Florida scientists create new approach to destroy disease-associated RNAs in cells
2012-12-21
(Press-News.org) 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.



INFORMATION:

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.


[Attachments] See images for this press release:
Scripps Florida scientists create new approach to destroy disease-associated RNAs in cells

ELSE PRESS RELEASES FROM THIS DATE:

WCS applauds Dept. of Interior plan balancing conservation and energy development in NPR-A

WCS applauds Dept. of Interior plan balancing conservation and energy development in NPR-A
2012-12-21
The Wildlife Conservation Society (WCS) lauded U.S. Secretary of the Interior Ken Salazars announcement of a final management plan for the National Petroleum Reserve-Alaska (NPR-A) that balances wildlife conservation and energy development in the biggest public landscape in the country. The Integrated Activity Plan and Final Environmental Impact Statement (Final IAP/EIS) issued today by the Bureau of Land Management is the first comprehensive land management plan ever developed for the NPR-A. By protecting extensive coastal plain habitat around Teshekpuk Lake, and the ...

Black piranha, megapiranha have most powerful bites of fish living or extinct, finds GW researcher

Black piranha, megapiranha have most powerful bites of fish living or extinct, finds GW researcher
2012-12-21
WASHINGTON— The black piranha and the extinct giant piranha, or megapiranha, have the most powerful bites of carnivorous fishes, living or extinct, once body size is taken into account, finds researchers in a paper recently published in Scientific Reports. The research paper, Mega-Bites: Extreme jaw forces of living and extinct piranhas, highlights the piranhas' specialized jaw morphology, which allows them to attack and bite chunks out of much larger prey. Guillermo Ortí, the George Washington University Louis Weintraub Professor of Biology in the Columbian College of ...

Transplanted genetically-modified adipose cells offer potential therapy for liver diseases

2012-12-21
Putnam Valley, NY. (Dec. 20, 2012) – Using mesenchymal stromal cells derived from adipose (fat) tissues, genetically modified to express a bioluminescent marker, researchers in Italy have tracked cells after transplantation. The cells were followed from their injection into the spleen of mice modeling liver disease, to their characterization as "hepatic precursors," and to their subsequent migration through the spleen before engrafting at regenerating sites in the liver by bioluminescent imaging. Their study is described in a recent issue of Cell Transplantation (21:9), ...

Cell Transplantation study investigates fate and function of cells transplanted to the CNS

2012-12-21
Putnam Valley, NY. (Dec. 20, 2012) – When different types of cells are transplanted with the intent of having them aid in repairing central nervous system (CNS) trauma, what is the fate and function of those cells? A Belgian research team carried out research aimed at answering this question by determining how five varieties of cells - neural stem cells, mouse embryonic fibroblasts, dendritic cells, bone marrow mononuclear cells (BMMNCs) and splenocytes - functioned and survived after transplantation in the CNS. Their study is published in a recent issue of Cell Transplantation ...

Study reports iron oxide nanoparticles effective for labeling human endothelial cells

2012-12-21
Putnam Valley, NY. (Dec. 20, 2012) – A team of researchers from three medical institutions in Guangzhou, China, have found that iron oxide nanoparticles (INOPS) are a useful contrast agent for in vivo magnetic resonance tracking of transplanted human endothelial cells. However, the impact of INOPS on the cells varies with a number of factors including the INOPS load. They found that the percentage of iron-labeled cells was significantly lower after 48 hours post-transplantation than at 24 hours post-transplantation. They also found that high INOPS concentration can affect ...

Doing the math for how songbirds learn to sing

2012-12-21
Scientists studying how songbirds stay on key have developed a statistical explanation for why some things are harder for the brain to learn than others. "We've built the first mathematical model that uses a bird's previous sensorimotor experience to predict its ability to learn," says Emory biologist Samuel Sober. "We hope it will help us understand the math of learning in other species, including humans." Sober conducted the research with physiologist Michael Brainard of the University of California, San Francisco. Their results, showing that adult birds correct ...

Young scientist helps identify cause of widespread eye disease

2012-12-21
Branch retinal vein occlusion – blockage of the blood vessels that channel blood from the retina – is a common eye disease. A type of blood clot in the eye, the disease causes reduced vision, and people with the disease also typically have an increased risk of hypertension, diabetes and other serious conditions. A young scientist from the University of Copenhagen has made a significant contribution to finding the cause of the disease. Mette Bertelsen and her colleagues photographically verified the diagnosis of branch retinal vein occlusion in 1168 people. A team of ...

NJ Association of Verismo Opera to Hold Auditions for Boys to Perform in Gianni Schicchi

2012-12-21
The NJ Association of Verismo Opera is known for developing the stars of tomorrow. Artistic Director Lucine Amara announces boys, nine to eleven years of age, with a soprano voice are invited to audition for the role Gherardino in the grand, professional opera company's production of Giacomo Puccini's Gianni Schicchi that will be performed on Sunday, April 21 at the Bergen Performing Arts Center (bergenPAC) in Englewood, New Jersey. Open auditions will be held on January 7, 2013 from 4-7 p.m. at the bergenPAC in the Cabaret Room, 30 North Van Brunt Street. The application ...

Powerstone Property Management Reports Double Digit Growth in 2012

2012-12-21
PowerStone Property Management Inc. a full service Orange County, California based property management firm, today announced that in 2012 it has added twenty-two new Home Owner Associations to its client roster representing a 25% growth jump from the previous year. Its professional staff has also increased by 12% over last year with the addition of 5 new employees. Client additions include a mix of new condominium, master planned, single family, and commercial properties. "Out of the 22 or so associations we gained this year, most came in the 3rd and 4th quarters." ...

MediPurpose Donates babyLance Heelsticks to Matthew: 25 Ministries

2012-12-21
MediPurpose(tm), a master distributor and manufacturer of medical products, today announced that it donated 1900 cases of babyLance(tm) infant heelsticks to international humanitarian aid and disaster relief organization Matthew 25: Ministries. Working closely with U.S. hospitals, corporations, businesses and individuals, Matthew 25: Ministries has shipped an estimated 115 million pounds of donated humanitarian supplies to the needy in the United States of America and impoverished countries in Central and South America, the Caribbean, the Balkans and Africa. "MediPurpose's ...

LAST 30 PRESS RELEASES:

Sensing sickness

Cost to build multifamily housing in California more than twice as high as in Texas

Program takes aim at drinking, unsafe sex, and sexual assault on college campuses

Inability to pay for healthcare reaches record high in U.S.

Science ‘storytelling’ urgently needed amid climate and biodiversity crisis

KAIST Develops Retinal Therapy to Restore Lost Vision​

Adipocyte-hepatocyte signaling mechanism uncovered in endoplasmic reticulum stress response

Mammals were adapting from life in the trees to living on the ground before dinosaur-killing asteroid

Low LDL cholesterol levels linked to reduced risk of dementia

Thickening of the eye’s retina associated with greater risk and severity of postoperative delirium in older patients

Almost one in ten people surveyed report having been harmed by the NHS in the last three years

Enhancing light control with complex frequency excitations

New research finds novel drug target for acute myeloid leukemia, bringing hope for cancer patients

New insight into factors associated with a common disease among dogs and humans

Illuminating single atoms for sustainable propylene production

New study finds Rocky Mountain snow contamination

Study examines lactation in critically ill patients

UVA Engineering Dean Jennifer West earns AIMBE’s 2025 Pierre Galletti Award

Doubling down on metasurfaces

New Cedars-Sinai study shows how specialized diet can improve gut disorders

Making moves and hitting the breaks: Owl journeys surprise researchers in western Montana

PKU Scientists simulate the origin and evolution of the North Atlantic Oscillation

ICRAFT breakthrough: Unlocking A20’s dual role in cancer immunotherapy

How VR technology is changing the game for Alzheimer’s disease

A borrowed bacterial gene allowed some marine diatoms to live on a seaweed diet

Balance between two competing nerve proteins deters symptoms of autism in mice

Use of antifungals in agriculture may increase resistance in an infectious yeast

Awareness grows of cancer risk from alcohol consumption, survey finds

The experts that can outsmart optical illusions

Pregnancy may reduce long COVID risk

[Press-News.org] 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