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:9/2/2014)... most ideal source of nutrition for infants and ... the evolution and civilizations of human beings. Unlike ... number of bacterial species, including some opportunistic pathogens ... to scientists and physicians. , Indeed, the existence ... result of co-evolutionary and co-adaptive interactions between the ...
(Date:9/2/2014)... integrity? Tell the truth; everyone has a tipping point. We ... lie if the benefit is great enough. Now, scientists have ... that decision. , The result was published online this ... prefer to be honest, even if lying is beneficial," said ... at the Virginia Tech Carilion Research Institute , where ...
(Date:9/2/2014)... propensity for eating anything, including each other, camel crickets ... North Carolina State University finds that non-native camel cricket ... States. , "The good news is that camel crickets ... humans," says Dr. Mary Jane Epps, a postdoctoral researcher ... about the research. , The research stems from a ...
Breaking Biology News(10 mins):Are human breast milk microbiome 'neutral'? 2Are human breast milk microbiome 'neutral'? 3Scientists find possible neurobiological basis for tradeoff between honesty, self-interest 2Scientists find possible neurobiological basis for tradeoff between honesty, self-interest 3Scientists find possible neurobiological basis for tradeoff between honesty, self-interest 4Researchers find Asian camel crickets now common in US homes 2
... the triangle depicting how many servings of each food ... knows about MyPlate - the circle showing what a healthy ... United States Department of Agriculture (USDA) to help American consumers ... icon that prompts us to think about what,s on our ...
... gene therapy holds promise for the treatment of devastating genetic ... have been unclear. In a study online December 26th ... by Cell Press, researchers evaluated a patient with a genetic ... after he had undergone a gene therapy procedure as part ...
... neurodegenerative disorders, and drugs without significant side effects ... Institute of Synergon Consulting in Romania, led by ... is a transcription co-activator required for consolidation of ... same time involved in DNA replication and repair, ...
Cached Biology News:Who is using MyPlate? 2Gene therapy for human skin disease produces long-term benefits 2
(Date:9/2/2014)... about a billionth of a meter in size, are ... can benefit human health, as in some innovative early ... through viruses, air pollution, traffic emissions, cosmetics, sunscreen and ... in St. Louis, led by Lan Yang, PhD, the ... Systems Engineering, and their collaborators at Tsinghua University in ...
(Date:9/2/2014)... Scientists, including University of Oregon chemist Geraldine Richmond, ... of self-assembling, synthetic proteins called peptoid nanosheets that ... accomplishment -- detailed this week in a paper ... Proceedings of the National Academy of Sciences ... of the two-dimensional peptoid nanosheets that can be ...
(Date:9/2/2014)... 2, 2014 Persistence Market Research ... in Pharmaceutical, Biopharma and Life Sciences: North ... the global membrane technology market for pharmaceutical, biopharma and life sciences ... is expected to grow at a CAGR of 9.1% ... of USD 10,886.0 million in 2019. Browse ...
(Date:9/2/2014)... AVENTURA, Fla. , Sept. 2, 2014  Technology ... has announced that its wholly-owned subsidiary Renuell Int,l, Inc. ... National Aeronautics and Space Administration ("NASA") at the Johnson ... See photos: tapplic.com/houstonphotos/ ... stated, "We are very pleased to announce our latest, ...
Breaking Biology Technology:Engineers develop new sensor to detect tiny individual nanoparticles 2Engineers develop new sensor to detect tiny individual nanoparticles 3UO-Berkeley Lab unveil new nano-sized synthetic scaffolding technique 2Global Membrane Technology Market Will Reach $10,886.0 Million in 2019: Persistence Market Research 2Global Membrane Technology Market Will Reach $10,886.0 Million in 2019: Persistence Market Research 3Global Membrane Technology Market Will Reach $10,886.0 Million in 2019: Persistence Market Research 4Global Membrane Technology Market Will Reach $10,886.0 Million in 2019: Persistence Market Research 5Technology Applications International Corporation Subsidiary Enters Into Additional Exclusive Agreement with NASA at a Signing Ceremony at the Johnson Space Center In Houston, Texas 2Technology Applications International Corporation Subsidiary Enters Into Additional Exclusive Agreement with NASA at a Signing Ceremony at the Johnson Space Center In Houston, Texas 3
... that it,has received the European CE Mark (Conformite Europeene) ... CE Mark certifies,that the product has met EU health, ... Granted in the Netherlands, the,registration will allow the system ... countries around the world that recognize the CE Mark. ...
... Calif., Oct. 29 Cliniqa Corporation (San,Marcos, ... is pleased to,announce the acquisition of the ... Diego, CA), a wholly owned subsidiary of ... manufacturer of clinical,diagnostic products focusing on clinical ...
... to Focus on Sales Growth and Product ... BioLife Solutions,Inc. (OTC Bulletin Board: BLFS), a ... and cryopreservation media products for,cells, tissues, and ... product,manufacturing, warehousing, and customer order fulfillment to ...
Cached Biology Technology:Diamics Receives European CE Mark Approval for Its Pap-Map(TM) System for Cervical Cancer Screening 2Cliniqa Corporation Completes Asset Acquisition of Reagents Applications, Inc. 2BioLife Solutions Outsourcing Manufacturing, Warehousing, and Order Fulfillment to Bioserv Corporation 2BioLife Solutions Outsourcing Manufacturing, Warehousing, and Order Fulfillment to Bioserv Corporation 3