Navigation Links
Cold Spring Harbor Laboratory scientists devise potential approach to treat spinal muscular atrophy
Date:4/4/2008

remove some sections and splice the others back together before the RNA is used to make protein. For many genes, the various sections can be mixed and matched, so that a single gene produces more than one kind of protein. Once considered a curiosity, this alternative splicing, of the RNA strand is now viewed as common, says Krainer, who is an expert on the topic.

Alternative Ways of Splicing RNA

When he first learned about SMA, Dr. Krainer says, "I was extremely excited, because I realized that what we knew about splicing could be applied" to the disease. All the researchers needed to do was to alter the splicing of the second copy of the gene to include the missing piece.

Dr. Krainer and his team sought to change the splicing by introducing synthetic molecules, called antisense oligonucleotides or ASOs, that precisely match various sections of the RNA. They reasoned that if these molecules stuck to the right part of the RNA, they might redirect the splicing process in the desired way. The researchers injected promising ASOs into mice that had an added, human version of the SMN2 gene. As they had hoped, the gene produced much more of the RNA for the critical protein, including the section that is usually omitted, in tissues where the ASOs accumulate.

Before this approach can be tried in patients, several additional issues must be addressed. Researchers will also need to find out, for example, whether the ASOs really benefit growing animals with SMA and how and when they should be administered to affect the nervous system. Still, in contrast to approaches that change splicing patterns for many genes, Krainer expects the highly targeted ASOs may have fewer side effects.


'/>"/>

Contact: Jim Bono
bono@cshl.edu
516-367-8455
Cold Spring Harbor Laboratory
Source:Eurekalert

Page: 1 2

Related biology news :

1. Cold Spring Harbor Protocols features methods to screen genomes and analyze evolution
2. Springer expands publishing partnership with the Biomedical Engineering Society
3. Warmer springs mean less snow, fewer flowers in the Rockies
4. Springer author Carlos M. Duarte awarded Spanish science prize
5. RNA-based methods for developmental studies are featured in Cold Spring Harbor Protocols
6. Cold Spring Harbor Laboratory to play central role addressing key questions in plant biology
7. Hot springs microbes hold key to dating sedimentary rocks, researchers say
8. Humana Press journals now available on SpringerLink
9. Cold Spring Harbor Laboratorys lung cancer research program awarded $100,000 grant
10. Springer launches Tropical Plant Biology
11. Moms obesity during conception phase may set the stage for offsprings obesity risk
Post Your Comments:
*Name:
*Comment:
*Email:
(Date:3/16/2015)... Between 16 and 20 ... innovations in biometric identification at the CeBIT exhibition ...      (Photo: http://photos.prnewswire.com/prnh/20150316/735333 ) , ... at the forefront: the company,s all new BiometricGate ... Germany . BiometricGate is the first ...
(Date:3/11/2015)... --Research and Markets ( http://www.researchandmarkets.com/research/3p32qm/access_control ) has ... by Product, Application & By Geography - Global Forecast ... , This report predicts that the access ... 2020, with an estimated CAGR of 10.6%. ... such as contact cards & readers, contactless cards & ...
(Date:3/10/2015)... March 10, 2015  NXT-ID, Inc. (NASDAQ: ... "Company"), a biometric authentication company focused on the ... smart wallet has been named the "Number One ... Rethink Modern ( http://www.rethinkmodern.com/ ) is ... your home and lifestyle that have a unique function ...
Breaking Biology News(10 mins):DERMALOG Presents its Latest Biometric Innovations at the Hannover CeBIT Exhibition, 16 - 20 March 2015 2DERMALOG Presents its Latest Biometric Innovations at the Hannover CeBIT Exhibition, 16 - 20 March 2015 3Global Access Control (Cards & Readers, Biometrics, Controllers, Management Software) Market 2015-2020 - Analysis of the $10 Billion Market 2NXT-ID's Wocket Smart Wallet Named Number One Best Tech Gift for 2015 by Rethink Modern 2NXT-ID's Wocket Smart Wallet Named Number One Best Tech Gift for 2015 by Rethink Modern 3NXT-ID's Wocket Smart Wallet Named Number One Best Tech Gift for 2015 by Rethink Modern 4
... largest national park as his laboratory, a University of ... change how we monitor and protect the environment. ... Canada through the Ontario Genomics Institute to conduct research ... most valued ecosystems. Hajibabaei,s grant proposal tied for ...
... New research has revealed how infection by a parasitic fungus ... Camponotus leonardi ), causing them to become zombie-like and ... for the fungus. The multinational research team studied ants living ... describing the research will be published in the BioMed Central ...
... live high up in the rainforest canopy. When infected ... behaviour of the ants is dramatically changed. They become ... into dying at a spot that provides optimal conditions ... open access journal BMC Ecology , looks at ...
Cached Biology News:Prof to use DNA technology to prevent habitat loss 2Zombie ants have fungus on the brain, new research reveals 2Zombie ants have fungus on the brain 2
(Date:3/25/2015)... March 25, 2015 /PRNewswire/ - SQI Diagnostics Inc. ("SQI" or ... a result of recent discussions with certain shareholders representing ... the Company, after dealing with certain matters including the ... of shareholders on March 26, 2015 (the "Meeting") it ... of directors will take place when the Meeting reconvenes. ...
(Date:3/25/2015)... 25, 2015  S&P Capital IQ (MHFI) announced ... Report coverage on Abattis Bioceuticals Corp . ... is a specialty biotechnology company with capabilities through ... marketing proprietary ingredients, bio-similar compounds, patented equipment and ... North America . The company seeks to ...
(Date:3/25/2015)... CAMBRIDGE, Mass. and REYKJAVIK, ... which provides comprehensive capabilities for using the genome to create ... their publication of the largest studies of whole-genome data yet ... in Nature Genetics – are built ... variation, authored by a team of deCODE scientists led by ...
(Date:3/25/2015)... (PRWEB) March 25, 2015 Data ... Novel Mechanism of Action in Neuromuscular Diseases ... – Cytokinetics, Incorporated (Nasdaq: CYTK) announced the publication ... muscle troponin activator tirasemtiv in the journal Neurotherapeutics. ... IIa “Evidence of Effect” or hypothesis-generating clinical trial ...
Breaking Biology Technology:SQI Diagnostics Inc. Announces New Nominees to SQI Diagnostics Board and Adjournment of Meeting 2SQI Diagnostics Inc. Announces New Nominees to SQI Diagnostics Board and Adjournment of Meeting 3SQI Diagnostics Inc. Announces New Nominees to SQI Diagnostics Board and Adjournment of Meeting 4SQI Diagnostics Inc. Announces New Nominees to SQI Diagnostics Board and Adjournment of Meeting 5S&P Capital IQ Initiates Coverage on Abattis Bioceuticals Corp. in S&P Capital IQ Factual Stock Reports 2S&P Capital IQ Initiates Coverage on Abattis Bioceuticals Corp. in S&P Capital IQ Factual Stock Reports 3S&P Capital IQ Initiates Coverage on Abattis Bioceuticals Corp. in S&P Capital IQ Factual Stock Reports 4S&P Capital IQ Initiates Coverage on Abattis Bioceuticals Corp. in S&P Capital IQ Factual Stock Reports 5Landmark deCODE Studies Demonstrate the Power of Truly Big Genomics for Improving Medicine Worldwide 2Cytokinetics Announces Publication of Results from Phase II Trial of Tirasemtiv in Patients with Myasthenia Gravis 2Cytokinetics Announces Publication of Results from Phase II Trial of Tirasemtiv in Patients with Myasthenia Gravis 3Cytokinetics Announces Publication of Results from Phase II Trial of Tirasemtiv in Patients with Myasthenia Gravis 4Cytokinetics Announces Publication of Results from Phase II Trial of Tirasemtiv in Patients with Myasthenia Gravis 5Cytokinetics Announces Publication of Results from Phase II Trial of Tirasemtiv in Patients with Myasthenia Gravis 6
... , D Garteiz, TexMS Analytical Services, ... , A Varian 1200L LC/MS/MS system ... topical delivery of therapeutic agents to the urinary tract using a urethra ... Background Urinary tract disease is a major health problem in the ...
... , BD Biosciences ... T Lymphocyte Separation Set DM (Cat. No. 558142) ... mononuclear cells (PBMC). The reagent set isolates Treg cells ... BD IMag streptavidin particles with a cocktail of biotinylated ...
... Cytokines are the hormonal messengers ... system, such as cell-mediated immunity and allergic type responses. T ... antigen-specific receptors on their cell surface to allow recognition of ... distinguished by the presence of cell surface molecules known as ...
Cached Biology Technology:Quantitation of Oxybutynin in Rabbit Plasma Using the Varian 1200L LC/MS/MS System 2Quantitation of Oxybutynin in Rabbit Plasma Using the Varian 1200L LC/MS/MS System 3Th1 and Th2 Balance, Regulation, and Involvement in Disease 2
... System brings together two mature, well-understood ... capture surfaces. This combination produces a ... the detection of molecular binding events ... , Protein-specific capture molecules are immobilized ...
... polymers) are prepared by ... the enzyme, polynucleotide phosphorylase, ... the appropriate, ribonucleoside diphosphate ... exception of catalog number ...
... comprehensive experiment based analysis package designed with ... sophisticated and powerful functionality., Image acquisition interface ... for easy image capture. Sequential and ... Playback Saturation Warning ...
...
Biology Products: