Navigation Links
Drug delivery strategy eliminates myotonia symptoms in mice with myotonic dystrophy
Date:2/22/2013

New Rochelle, NY, February 21, 2013By targeting the specific mutation that causes the hereditary neuromuscular disease myotonic dystrophy, it is possible to neutralize the mutant RNA toxicity and minimize or even eliminate the disabling symptoms of the disease. New classes of drugs called antisense oligonucleotides are being designed to achieve this. Innovative work to develop a modified antisense drug that can be administered intravenously and achieve the desired therapeutic effect is described in an article in Nucleic Acid Therapeutics, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available free on the Nucleic Acid Therapeutics website.

Andrew Leger and coauthors from Genzyme, a Sanofi Company (Framingham, MA) added a peptide to an oligonucleotide strand designed to bind to and inactivate the mutated RNA region associated with myotonic dystrophy type 1 (DM1). The disease can affect function of the heart, central nervous system, and gastrointestinal tract, and a characteristic symptom is myotonia, in which muscles are slow to relax following contraction.

In the article "Systemic Delivery of Peptide-Linked Morpholino Oligonucleotide Neutralizes Mutant RNA Toxicity in a Mouse Model of Myotonic Dystrophy," the authors describe how the peptide is intended to enable systemic delivery of the drug, protecting it from being damaged or destroyed in the body before it can reach its target, the muscles. They report that intravenous introduction of the drug in a mouse model of DM1 led to good biodistribution of the drug, evidence that the problems previously caused by RNA toxicity were corrected, and complete elimination of myotonia in the treated mice.

"One of the greatest challenges to the
'/>"/>

Contact: Vicki Cohn
vcohn@liebertpub.com
914-740-2100 x2156
Mary Ann Liebert, Inc./Genetic Engineering News
Source:Eurekalert  

Page: 1 2

Related biology news :

1. Ben-Gurion U. licenses drug delivery platform for central nervous system diseases to NY biotech firm
2. Delivery of gene-therapy for heart disease boosted 100-fold; now in 100-patient trial
3. Light-induced delivery of nitric oxide eradicates drug-resistant bacteria
4. Oligonucleotide Delivery: Biology, Engineering and Development Conference
5. Elegant delivery
6. Pilot Results Move GHX Closer to Delivery of Healthcare Industrys First Implantable Device Supply Chain Solution
7. University of Leicester takes delivery of unique eye imaging equipment
8. Body heat, fermentation drive new drug-delivery micropump
9. UC Davis researchers develop new drug delivery system for bladder cancer using nanoparticles
10. Aware Enables Automated Delivery of Radiation Dose Information Through Integration With Leading Radiology Reporting System
11. Nanotechnology drug delivery shows promise for treatment of pediatric cancer
Post Your Comments:
*Name:
*Comment:
*Email:
Related Image:
Drug delivery strategy eliminates myotonia symptoms in mice with myotonic dystrophy
(Date:9/30/2014)... it comes to using marijuana, new research, involving mice ... Journal of Leukocyte Biology , suggests ... that you should. That,s because a team of Italian ... do serious long-term damage to the immune system. This ... diseases, such as multiple sclerosis, inflammatory bowel disease and ...
(Date:9/30/2014)... Mass. , Sept. 30, 2014 /PRNewswire/ ... control solutions for the biotech and power ... its new RECONN Bioprocess Manager, an easy-to-use ... solution geared towards bioprocess development laboratories. ... have disparate bioreactor and analyzer systems. Accessing ...
(Date:9/29/2014)... exposure to tar tended to be lower for smokers ... exposure to nicotine tended to be lower. , Slim ... several countries around the world. Previous studies have shown ... smoke of these cigarettes are lower than those in ... the smoke are not necessarily linked to a reduced ...
Breaking Biology News(10 mins):Adolescent exposure to thc may cause immune systems to go up in smoke 2Spectra Automation Introduces RECONN Bioprocess Manager 2Slim cigarette smokers not exposed to more harmful chemicals 2
... the Arctic is boosted by global warming, it,s not enough ... permafrost beneath the surface, University of Florida researchers have found ... thawing of permafrost in a warming world. Twice as ... the atmosphere today, and what happens to it as it ...
... the first time in mammalian brain cells, researchers at ... in building the circuitry that relays signals through the ... aging process in the brain, the researchers say. Additionally, ... discovery paves the way for faster progress toward identifying ...
... back on a shark" is the take home message from ... Erich Ritter of the Shark Research Institute and Raid Amin ... that sharks can comprehend body orientation and therefore know whether ... to approach and possibly attack their prey from the blind ...
Cached Biology News:UF researchers' experiment is first to simulate warming of Arctic permafrost 2UF researchers' experiment is first to simulate warming of Arctic permafrost 3Gene found to be crucial for formation of certain brain circuitry 2Sharks prefer to sneak up from behind, study shows 2
(Date:10/1/2014)... , Oct. 1, 2014  Spherix Incorporated (Nasdaq: ... fostering and monetization of intellectual property, announced today ... VTech and Uniden in the U.S. District Court ... has been rescheduled for November 10, ... 2, 2014.  The Court Order setting the Markman ...
(Date:10/1/2014)... In response to its clients’ ... sales, training and consumer engagement challenges, digital ... applications to turn their clients’ visions into reality. ... mobile app creations, are now featured on its ... with Toyota, StudioPMG created a robust cross-platform mobile ...
(Date:10/1/2014)... The U.S. Department of Labor has awarded a ... part of the Trade Adjustment Assistance Community College and ... the Department of Labor and Department of Education. , ... Perez and Secretary of Education Arne Duncan announced the ... nearly 270 colleges across the country. , The ...
(Date:9/30/2014)... German . ... have demonstrated a new kind of building block for ... chips could be based on three-dimensional arrangements of nanometer-scale ... of the semiconductor industry CMOS fabrication of silicon ... collaborators at the University of Notre Dame are exploring ...
Breaking Biology Technology:Markman Hearing Date for VTech and Uniden Litigation Now Set for November 10, 2014 2Digital Agency Studio PMG Launches New Case Studies on Website 2U.S. Dept. of Labor Awards SIUE Nearly $10-Million Grant 2U.S. Dept. of Labor Awards SIUE Nearly $10-Million Grant 3U.S. Dept. of Labor Awards SIUE Nearly $10-Million Grant 4A new dimension for integrated circuits: 3-D nanomagnetic logic 2A new dimension for integrated circuits: 3-D nanomagnetic logic 3
... HAYWARD, Calif., Jan. 12 Anthera Pharmaceuticals, Inc., ... that it has completed enrollment in its 500 ... Cardiovascular,Inflammation Suppression) clinical trial designed to examine the ... 96 hours of an Acute,Coronary Syndrome (ACS) event. ...
... Md., Jan. 9 Human Genome Sciences, Inc. (Nasdaq: ... at the 27th Annual JPMorgan Healthcare Conference will be ... http://www.newscom.com/cgi-bin/prnh/20080416/HGSLOGO )A member of Human Genome ... the company on Wednesday, January 14, 2009 at 7:30 ...
... NS, Jan. 9 /PRNewswire/ - ImmunoVaccine Technologies Inc. (IVT), ... present at the OneMedPlace Finance Forum on Tuesday, January ... visit www.onemedplace.com . , Dr. Randal Chase, president ... 8:45 am PST on the development of its superior ...
Cached Biology Technology:Anthera Completes Enrollment and Dosing of Varespladib Cardiovascular Trial 2Anthera Completes Enrollment and Dosing of Varespladib Cardiovascular Trial 3
Required to perform reference file generation, flatfield calibration and performance verification processes on the GSD-501...
ssDNA from PCR DNA in less than 1 hour. No gels, columns, or slurries. Sequence from high quality ssDNA: clearer reads, longer runs....
... of Freezerworks Unlimited includes 2 clients and streamlines ... the sharing of freezers by multiple groups without ... Network Client/Server version for ... monitor with 256 colors; OS 8.6 & OS ...
Applications: Immunohistochemistry, ISH, FISH, CISH....
Biology Products: