Navigation Links
Silence the gene, save the cell: RNA interference as promising therapy for ALS

research also indicates that mutant SOD1 gene expression in neighboring glial cells is also implicated in motor neuron death.

Lead author Cedric Raoul and colleagues targeted the cause of the disease by using RNA interference to silence the defective gene, preventing it from expressing the SOD1 protein.

RNA interference is part of a complex cellular housekeeping process that protects cells from invading viruses or other genetic threats. It works by interrupting messenger RNA as it transfers the genetic code for a protein from the nucleus to the site in the cell where the protein is synthesized.

To trigger RNA interference and silence a gene, short bits of double-stranded RNA are introduced in the cell, where they bind with matching sections of messenger RNA. The cell identifies the resulting messenger RNA strand as faulty and chops it up. As a result, the genetic blueprint isn't delivered and the protein never gets made.

"Gene silencing is an example of using "molecular scissors" at its most advanced level," Raoul explains.

Raoul and colleagues used RNA interference to reduce levels of mutant SOD1 protein in the spinal cords of transgenic ALS mice (mice bred to express the human SOD1 gene). Short strands of RNA that targeted multiple mutated and normal forms of the human SOD1 gene were delivered in a specially engineered lentivirus. Expression of the SOD1 protein was knocked down in the affected motor neurons and neighboring glial cells, and both the onset and the rate of progression of the disease in the treated mice were substantially reduced. In addition, the mice showed a significant improvement in neuromuscular function.

"This is the first demonstration of therapeutic efficacy in vivo of RNA interference-mediated gene silencing in an ALS model," notes Raoul.

Because the normal form of the SOD1 protein may be necessary for the survival or function of adult human motor neurons, the Swiss researchers designed
'"/>

Source:Ecole Polytechnique F茅d茅rale de Lausanne


Page: 1 2 3

Related biology news :

1. Silence of the amoebae
2. Markers of gene, protein, or micro-RNA activity predict outcome in prostate and colorectal cancers
3. Microbial fuel cell: High yield hydrogen source and wastewater cleaner
4. Scientists identify genetic pathways essential to RNA interference
5. Scientists identify genetic pathways essential to RNA interference
6. Used in a new way, RNA interference permanently silences key breast cancer gene
7. RNA interference stops colon cancer spread in mice
8. Cocaine high caused by interference in neuronal receiving stations
9. Gene vaccine for Alzheimers disease shows promising results
10. Gene therapy promising for growing tooth-supporting bone
11. Researchers find promising cancer-fighting power of synthetic cell-signaling molecule
Post Your Comments:
(Date:9/18/2014)... researchers from McGill University and the Quebec government ... ,microbeads, Canadian Journal of Fisheries and Aquatic ... cosmetics, household cleansers, or industrial cleansers, to which ... their small size and buoyancy, they may readily ... a global contaminant in the world,s oceans, but ...
(Date:9/18/2014)... No matter how many times it,s demonstrated, it,s ... creatures. , But by using a signaling ... alter their behavior to suit their population. In ... are present, and act accordingly. , Once the ... may change from innocuous to pathogenic, or from ...
(Date:9/18/2014)... language is unique to humans. "The genetic changes that ... evolution to make this possible are largely unknown, but ... says Wolfgang Enard, Professor of Anthropology and Human Biology ... biological basis of language Enard has now taken an ... undertaken in collaboration with scientists at several universities, including ...
Breaking Biology News(10 mins):Microplastic pollution discovered in St. Lawrence River sediments 2Researchers study vital 'on/off switches' that control when bacteria turn deadly 2Researchers study vital 'on/off switches' that control when bacteria turn deadly 3Language evolution: Quicker on the uptake 2Language evolution: Quicker on the uptake 3
... University Feinberg School of Medicine played a significant ... Abraxane (paclitaxel protein-bound particles for injectable suspension), indicated ... approval means that women with metastatic breast cancer ... with solvents and will no longer need steroid ...
... In laboratory mouse experiments, researchers at The University of ... way to use RNA interference (RNAi) so that it ... activated STAT3, a crucial gene found in some human ... invasive. , The study, presented at the annual meeting ...
... provide for study of human immune responses, scientists have ... rapid immune response by two separate armies of defensive ... microbes like the leprosy bug, this initial defense sometimes ... yields to a dangerous, spreading infection. , Led by ...
Cached Biology News:Nanoparticle Breast Cancer Drug Approved by FDA 2Used in a new way, RNA interference permanently silences key breast cancer gene 2Leprosy microbes lead scientists to immune discovery 2
(Date:9/19/2014)... California (PRWEB) September 19, 2014 ... and its potential to revolutionize the world has ... multinational corporations and small start-ups. The early years ... research projects largely due to the high risks ... Government budgets earmarked for this evolving technology have ...
(Date:9/19/2014)... SAN FRANCISCO , Sept. 19, 2014 /PRNewswire/ ... announced results from preclinical studies characterizing the analgesic ... oral, peripherally-acting kappa opioid receptor agonist molecules. The ... molecule polymer medicinal chemistry platform. The ... described in the medical literature. 1,2 Kappa ...
(Date:9/19/2014)... 19, 2014 Barnhardt Manufacturing Company ... for its Organic Cotton . The USDA Certified ... renewable biobased ingredients meets or exceeds levels set by ... in whole or in significant part of agricultural, forestry, ... our Organic cotton to be immediately identified as a ...
(Date:9/19/2014)... -- , The first grants awarded to ... (ESPE) Meeting  Investment supports innovative projects for ... field of growth  , EMD Serono, a ... , today announced the first recipients of the Grant ... announced during a Satellite Symposium organized by EMD Serono ...
Breaking Biology Technology:Growing R&D Investments & Developments in Nanomaterials to Drive the Global Nanotechnology Market, According to New Report by Global Industry Analysts, Inc. 2Growing R&D Investments & Developments in Nanomaterials to Drive the Global Nanotechnology Market, According to New Report by Global Industry Analysts, Inc. 3Growing R&D Investments & Developments in Nanomaterials to Drive the Global Nanotechnology Market, According to New Report by Global Industry Analysts, Inc. 4Nektar Presents Positive Preclinical Data on Oral, Peripherally-Acting Kappa Agonist Molecules at 2014 American Academy of Pain Management Meeting 2Nektar Presents Positive Preclinical Data on Oral, Peripherally-Acting Kappa Agonist Molecules at 2014 American Academy of Pain Management Meeting 3Nektar Presents Positive Preclinical Data on Oral, Peripherally-Acting Kappa Agonist Molecules at 2014 American Academy of Pain Management Meeting 4Nektar Presents Positive Preclinical Data on Oral, Peripherally-Acting Kappa Agonist Molecules at 2014 American Academy of Pain Management Meeting 5Barnhardt Manufacturing Company Earns USDA Certified Biobased Product Certification and Label for Organic Cotton 2EMD Serono Awards Grant for Growth Innovation (GGI) for the First Time 2EMD Serono Awards Grant for Growth Innovation (GGI) for the First Time 3
... , , HUNTERSVILLE, N.C., ... leader in built-in antimicrobial product protection, has partnered with YogaDirect.com, ... the U.S., to launch the first ever line of yoga ... of yoga mats that will stay cleaner and fresher for ...
... , DOYLESTOWN, Pa., July 28 ... company to offer a broad range of accredited CME courses in ... clinical trials. , , The comprehensive education and ... Institute (BRITI)(TM). RadMD is partnering with International Center for Postgraduate Medical ...
... , , WESTFORD, Mass., July ... manufacturer of a broad array of light-based aesthetic treatment systems, today announced financial ... Second Quarter 2009 Financial Results , , ... with $39.2 million in the same period of 2008 and $14.8 million in ...
Cached Biology Technology:Microban Partners With YogaDirect.com to Launch Yoga Mats With Zinc Antimicrobial Technology 2RadMD First to Offer CME-Certified Education and Training For Medical Imaging in Clinical Trials 2Cynosure Reports Second Quarter 2009 Financial Results 2Cynosure Reports Second Quarter 2009 Financial Results 3Cynosure Reports Second Quarter 2009 Financial Results 4Cynosure Reports Second Quarter 2009 Financial Results 5Cynosure Reports Second Quarter 2009 Financial Results 6Cynosure Reports Second Quarter 2009 Financial Results 7Cynosure Reports Second Quarter 2009 Financial Results 8Cynosure Reports Second Quarter 2009 Financial Results 9Cynosure Reports Second Quarter 2009 Financial Results 10Cynosure Reports Second Quarter 2009 Financial Results 11Cynosure Reports Second Quarter 2009 Financial Results 12Cynosure Reports Second Quarter 2009 Financial Results 13
... The four-bay DNA Engine Tetrad ... of interchangeable Alpha units (sample-holder, heat-pump ... numbers of vessels. A Pentium-class computer ... displays current block, sample, and lid ...
Printer paper for Model 680 series readers is used with the thermal printers of the Model 680 and Model 680 XR microplate readers. Supplied as 2 rolls....
... for 2-D separations. The cell has a six-gel ... xi multi-cell can be customized for several parameters: ... cooling system. The PROTEAN II xi multi-cell can ... multi-cell for a wider format that accommodates 17 ...
... is tailored to highly enrich ... rat spleen cells. Cells are ... compatible 0.5 Tesla) immunomagnetic negative ... content of the enriched fraction ...
Biology Products: