Navigation Links
Scripps Florida scientists design molecule that reverses some fragile X syndrome defects
Date:9/4/2012

JUPITER, FL - Scientists on the Florida campus of The Scripps Research Institute have designed a compound that shows promise as a potential therapy for one of the diseases closely linked to fragile X syndrome, a genetic condition that causes mental retardation, infertility, and memory impairment, and is the only known single-gene cause of autism.

The study, published online ahead of print in the journal ACS Chemical Biology September 4, 2012, focuses on tremor ataxia syndrome, which usually affects men over the age of 50 and results in Parkinson's like-symptomstrembling, balance problems, muscle rigidity, as well as some neurological difficulties, including short-term memory loss and severe mood swings.

With fragile X syndrome, tremor ataxia syndrome, and related diseases, the root of the problem is a structural motif known as an "expanded triplet repeat"in which a series of three nucleotides are repeated more times than normal in the genetic code of affected individuals. This defect, located in the fragile X mental retardation 1 (FMR1) gene, causes serious problems with the processing of RNA.

"While there is an abundance of potential RNA drug targets in disease, no one has any idea how to identify or design small molecules to target these RNAs," said Mathew Disney, a Scripps Research associate professor who led the study. "We have designed a compound capable of targeting the right RNA and reversing the defects that cause fragile X-associated tremor ataxia."

Preventing Havoc

In tremor ataxia syndrome, the expanded triplet repeat leads to the expression of aberrant proteins that wreak widespread havoc. The repeats actually force the normal proteins that regulate RNA splicingnecessary for production of the right kind of proteinsinto hiding.

The compound designed by Disney and his colleagues not only improves the RNA splicing process, but also minimizes the ability of repeats to wreak havoc on
'/>"/>

Contact: Mika Ono
mikaono@scripps.edu
858-784-2052
Scripps Research Institute
Source:Eurekalert  

Page: 1 2

Related biology news :

1. Scripps Research discoveries lead to newly approved drug for infant respiratory distress syndrome
2. Scripps Research Institute scientists find promising vaccine targets on hepatitis C virus
3. Scripps Research Institute Professor Gerald F. Joyce elected to American Academy of Arts & Sciences
4. Scripps Research Institute scientists develop antidote for cocaine overdose
5. Scientist wins $3 million renewal of one of longest-running NIH grants to Scripps Research
6. Scripps research scientists find anticonvulsant drug helps marijuana smokers kick the habit
7. Scripps Florida scientist awarded $1.5 million to design therapeutics with new RNA approach
8. Scripps Florida scientists identify neurotranmitters that lead to forgetting
9. Plastic trash altering ocean habitats, Scripps study shows
10. Scripps Florida scientists awarded $8.4 million grant to develop new anti-smoking treatments
11. Esther B. OKeeffe Foundation gives $2 million to the Scripps Research Institute
Post Your Comments:
*Name:
*Comment:
*Email:
Related Image:
Scripps Florida scientists design molecule that reverses some fragile X syndrome defects
(Date:11/21/2014)...  Earlier this year Donald Spector , Chairman ... of the most prolific inventors in the world, got ... Smartphones to third party agencies. Spector envisioned this technology ... the earliest known patents in this area. Now major ... child care, elder care and hospital applications. This technology ...
(Date:11/21/2014)...   Atmel® Corporation (NASDAQ: ATML ... technology solutions, today launched the industry,s first family of ... V cc range from 1.7V to 5.5V. The ... 2 C bus communication speeds, and are available with ... ideal for consumer, industrial, computer, and medical applications. ...
(Date:11/18/2014)... , Nov. 17, 2014 The Parenteral Drug ... U.S. regulatory agencies will speak and at least seven more ... Conference at the Omni Shoreham Hotel in Washington ... once again to have significant support from the regulatory agencies ... Europe in our effort to help advance ...
Breaking Biology News(10 mins):Patented Biosensor Wearable Technology Provides a More Civilized Method of Quarantine 2Patented Biosensor Wearable Technology Provides a More Civilized Method of Quarantine 3Patented Biosensor Wearable Technology Provides a More Civilized Method of Quarantine 4Atmel Launches Industry's First Wide-V(cc) Low-Power Temperature Sensor Family 2Atmel Launches Industry's First Wide-V(cc) Low-Power Temperature Sensor Family 3Atmel Launches Industry's First Wide-V(cc) Low-Power Temperature Sensor Family 4FDA's Janet Woodcock, EMA's Emer Cooke Headline PDA Quality Metrics Conference 2
... have a well-earned reputation for being dopey. Sleeping 19 hours ... remaining 5 hours, there doesn,t seem to be much time ... the mating season. Then the males begin bellowing. Benjamin Charlton ... bellow to attract females and to intimidate other males. But ...
... structure rich, poor and sometimes middle instead of ... to Stanford University researchers, it is the very inequities of ... spread of those societies and the displacement of more egalitarian ... researchers used a computer simulation to compare demographic stability and ...
... NCForest Service Southern Research Station (SRS) scientists recently ... (Coweeta) in Western North Carolina to examine the ... the changing weather conditions forecast for the future. ... the journal Ecological Applications, the analysis examines the ...
Cached Biology News:Koalas' bellows boast about size 2Suffering of the poor may have helped societies with class structures spread across globe 2Managing future forests for water 2
(Date:11/22/2014)... CannLabs, Inc. (OTCQB: CANL), the leading ... methodologies relating to cannabis, today announced that it has ... from an existing stockholder of the Company. , ... from one of our existing stockholders,” stated Mark ... help accelerate our planned expansions into the Connecticut and ...
(Date:11/22/2014)... 2014  The element of surprise has been a ... strike without warning. Until now, epidemiologists had nothing to ... outbreak, resulting in suboptimal responses to both the H1N1 ... Photo - http://photos.prnewswire.com/prnh/20141121/160242 ... because virus gene structural changes have been found that ...
(Date:11/21/2014)... (PRWEB) November 20, 2014 Offering a ... Sartorius Entris Balance promotion . The Sartorius Entris ... a durable, high accuracy, and easy-to-maintain balance entry level ... Germany. It was designed to help customer’s bridge the ... a balance that is very durable. Sartorius is the ...
(Date:11/21/2014)... BlueInGreen, LLC ( BlueInGreen® ) ... and PCI, Inc., leading suppliers of ozone process ... selected to provide a new state-of-the-art ozone system ... Noland Wastewater Treatment. The new ozone disinfection ... gas dissolution technology, Pinnacle Ozone’s industry leading Zenith™ ...
Breaking Biology Technology:CannLabs Secures $750,000 Line Of Credit 2Viruses' Advantage of Surprise is Lost 2Pipette.com Announces an Extension on the Sartorius Entris Balance Promotion to the End of 2014 2City of Fayetteville Leads Nation with Innovative Ozone Technology 2City of Fayetteville Leads Nation with Innovative Ozone Technology 3City of Fayetteville Leads Nation with Innovative Ozone Technology 4
... RIDGE, Tenn., July 19, 2010 -- Tomorrow,s television and ... as a result of a process developed by a ... Energy,s Oak Ridge National Laboratory. The synthesis of ... in devices like light-emitting diodes, televisions and solar cells--could ...
... new market research report is available in its catalogue: , , ... Applications. 2009-2015 , , , ... , REPORT DESCRIPTION , ... The ,Stem Cells Market and Applications: (2009-2015), reviews the latest Stem Cells market trends with ...
... create genetically altered mosquitoes that cannot infect humans ... mosquitoes still were capable of transmitting the disease-causing ... for the first time, University of Arizona entomologists ... way that renders them completely immune to the ...
Cached Biology Technology:Polymer synthesis could aid future electronics 2Reportlinker Adds Stem Cells Market And Applications. 2009-2015 2Reportlinker Adds Stem Cells Market And Applications. 2009-2015 3Reportlinker Adds Stem Cells Market And Applications. 2009-2015 4Reportlinker Adds Stem Cells Market And Applications. 2009-2015 5Reportlinker Adds Stem Cells Market And Applications. 2009-2015 6Reportlinker Adds Stem Cells Market And Applications. 2009-2015 7Reportlinker Adds Stem Cells Market And Applications. 2009-2015 8Reportlinker Adds Stem Cells Market And Applications. 2009-2015 9Reportlinker Adds Stem Cells Market And Applications. 2009-2015 10Reportlinker Adds Stem Cells Market And Applications. 2009-2015 11Reportlinker Adds Stem Cells Market And Applications. 2009-2015 12Reportlinker Adds Stem Cells Market And Applications. 2009-2015 13Reportlinker Adds Stem Cells Market And Applications. 2009-2015 14Reportlinker Adds Stem Cells Market And Applications. 2009-2015 15Reportlinker Adds Stem Cells Market And Applications. 2009-2015 16Reportlinker Adds Stem Cells Market And Applications. 2009-2015 17Reportlinker Adds Stem Cells Market And Applications. 2009-2015 18Reportlinker Adds Stem Cells Market And Applications. 2009-2015 19Reportlinker Adds Stem Cells Market And Applications. 2009-2015 20Reportlinker Adds Stem Cells Market And Applications. 2009-2015 21Reportlinker Adds Stem Cells Market And Applications. 2009-2015 22Reportlinker Adds Stem Cells Market And Applications. 2009-2015 23Reportlinker Adds Stem Cells Market And Applications. 2009-2015 24Reportlinker Adds Stem Cells Market And Applications. 2009-2015 25Reportlinker Adds Stem Cells Market And Applications. 2009-2015 26Reportlinker Adds Stem Cells Market And Applications. 2009-2015 27Reportlinker Adds Stem Cells Market And Applications. 2009-2015 28Reportlinker Adds Stem Cells Market And Applications. 2009-2015 29Reportlinker Adds Stem Cells Market And Applications. 2009-2015 30Reportlinker Adds Stem Cells Market And Applications. 2009-2015 31Reportlinker Adds Stem Cells Market And Applications. 2009-2015 32Reportlinker Adds Stem Cells Market And Applications. 2009-2015 33The first malaria-proof mosquito 2The first malaria-proof mosquito 3
... 0.15M NaCl, 20mM MOPS, pH 7.5, ... 1mM EGTA, 10 Properites: For selective ... DEAE chromatography, divalent cation activation plus ... Quality Assurance: routinely evaluated by dephosphorylation ...
Mouse monoclonal [6G11] to Cytohesin 1 + 2 ( Abpromise for all tested applications)....
HNF-1 Purified Anti-Mouse, Anti-Rat, Anti-Rabbit, Anti-Human clone 2, Isotype Mouse IgG 1 , 50 µg Consult technical datasheet for details....
Recombinant Porcine IL-1 alpha/IL-1F1, CF...
Biology Products: