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:7/24/2014)... ply silent freeways, solar panels blanket rooftops and power ... howling winds and from the blazing desert sun. , ... and economically feasible to convert California,s all-purpose energy infrastructure ... Energy , the plan shows the way to ... that could create tens of thousands of jobs and ...
(Date:7/24/2014)... at Albert Einstein College of Medicine of ... J. Fox Foundation for Parkinson,s Research to translate a ... This drug discovery project will test chemical compounds on ... a drug that acts on an underlying cause of ... help many people manage their symptoms, we are eager ...
(Date:7/24/2014)... hazard in the United States, wildland managers often utilize ... suppressed trees (ladder fuels). These cuttings and other ... fire danger in order to dispose of the material. ... managers often cover all or part of the debris ... agricultural plastic, in order to keep water out. A ...
Breaking Biology News(10 mins):Stanford study shows how to power California with wind, water and sun 2Stanford study shows how to power California with wind, water and sun 3Stanford study shows how to power California with wind, water and sun 4Albert Einstein College of Medicine receives grant from the Michael J. Fox Foundation to fund drug discovery project targeting Parkinson's 2Albert Einstein College of Medicine receives grant from the Michael J. Fox Foundation to fund drug discovery project targeting Parkinson's 3Study gives new perspective on agricultural plastic, debris burning, and air quality 2
... the National Consortium for Data Science (NCDS) aims to ... and data science research, a move that will help ... the data-driven global economy. The consortium, ... University of North Carolina at Chapel Hill, unites data ...
... reveals that age matters when it comes to adapting to ... insight and understanding of the likely impact of predicted environmental ... the journal Global Change Biology scientists from British ... the Alfred Wegener Institute reveal that when it comes to ...
... An international team of researchers has decoded the genome ... and illuminating: the African coelacanth. A sea-cave dwelling, five-foot ... thought to be extinct. A living coelacanth was discovered ... questions about these ancient-looking fish popularly known as ...
Cached Biology News:National consortium to lead North Carolina in big data innovation 2National consortium to lead North Carolina in big data innovation 3Age matters to Antarctic clams 2Coelacanth genome surfaces 2Coelacanth genome surfaces 3Coelacanth genome surfaces 4
(Date:7/24/2014)... Texas (PRWEB) July 24, 2014 ... Potassium Sulphate Industry” is a professional and in-depth ... report introduces Potassium Sulphate basic information, including ... and industry overview. This research covers the international ... as global industry analysis covering macroeconomic environment & ...
(Date:7/24/2014)... Draper University, a top program ... announce that for 6 weeks this summer, 39 ... on the Draper University campus in the heart ... focused on entrepreneurship, building real-world skills, and “learning ... its sixth cohort, continues to refresh its curriculum ...
(Date:7/23/2014)... , July 24, 2014 Lyfebulb, ... Pharmaceuticals Inc. (OTCQX and NASDAQ OMX First North ... or "the Company"), announced a collaboration to increase ... last night at an event attended by more ... parties. Lyfebulb is a health and wellness company ...
(Date:7/23/2014)... the Helmholtz-Zentrum Dresden-Rossendorf (HZDR), the Vienna University of ... succeeded in embedding nearly perfect semiconductor crystals into ... producing hybrid nanowires, very fast and multi-functional processing ... in the future. The research results will be ... , Nano-optoelectronics are considered the cornerstone of future ...
Breaking Biology Technology:Potassium Sulphate Market & SHMP Industry 2014 (Global, China) Analysis Now Available at DeepResearchReports.com 2Potassium Sulphate Market & SHMP Industry 2014 (Global, China) Analysis Now Available at DeepResearchReports.com 3Potassium Sulphate Market & SHMP Industry 2014 (Global, China) Analysis Now Available at DeepResearchReports.com 4Summer Entrepreneurship Program Kicked Off by Draper University in Silicon Valley 2Summer Entrepreneurship Program Kicked Off by Draper University in Silicon Valley 3Innovative Collaboration To Increase Awareness Of Bullous Pemphigoid, A Rare Autoimmune Disease Of The Skin 2Innovative Collaboration To Increase Awareness Of Bullous Pemphigoid, A Rare Autoimmune Disease Of The Skin 3Innovative Collaboration To Increase Awareness Of Bullous Pemphigoid, A Rare Autoimmune Disease Of The Skin 4Innovative Collaboration To Increase Awareness Of Bullous Pemphigoid, A Rare Autoimmune Disease Of The Skin 5A crystal wedding in the nanocosmos 2
... Microsoft chairman and chief software architect Bill Gates talked ... industry when he stopped in their classroom during his ... included in the tour, which is promoting greater youth ... Communications. Photo by Jeff Miller , ...
... Wis. - These are not your parents' generation ... "cool" was rarely heard to cross the lips of ... commodity. The problem is that Wisconsin doesn't yet have ... dozen representatives of business, civic, academic and arts groups ...
... Madison, Wis. - The 53 Wisconsin companies that ... Business Innovation Research (SBIR) and Small Business Technology Transfer ... recognized at an upcoming banquet in Madison. , ,The ... technology transfer workshop. The workshop will focus on SBIR/STTR ...
Cached Biology Technology:Gates displays software's future to UW-Madison students 2Gates displays software's future to UW-Madison students 3Gates displays software's future to UW-Madison students 4Tech leaders call for more creativity 2
Presenilin 2 Immunogen: Synthetic peptide corresponding to residues 31-47 of human Presenilin 2. Storage: -20 C, Avoid Freeze/Thaw Cycles...
Monoclonal Anti-Gα o purified immunoglobulin buffered aqueous solution Solution in phosphate buffered saline pH 7.4, containing 0.08% sodium azide. partially purified bovine brain Gα O ...
Potassium Channel kv3.2...
... 2, Neuronal (GLYT2). The antiserum has been ... nervous system. The staining pattern obtained with ... in situ hybridization with probes to GLYT2 ... for glycinergic neurons. Preabsorption of the antiserum ...
Biology Products: