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 therapeutic use of nucleic acids is effective and safe delivery," says Executive Editor Fintan Steele, PhD, SomaLogic, Inc., Boulder, CO. "The work of Leger and his colleagues demonstrates a potentially powerful way to meet that challenge for many diseases."


'/>"/>

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

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:5/12/2016)... , May 12, 2016 WearablesResearch.com , ... published the overview results from the Q1 wave of ... recent wave was consumers, receptivity to a program where ... with a health insurance company. "We were ... share," says Michael LaColla , CEO of Troubadour ...
(Date:4/28/2016)... BANGALORE, India , April 28, 2016 ... subsidiary of Infosys (NYSE: INFY ), and Samsung ... global partnership that will provide end customers with a ... and payment services.      (Logo: http://photos.prnewswire.com/prnh/20130122/589162 ... for financial services, but it also plays a fundamental part ...
(Date:4/26/2016)... -- Research and Markets has announced the ...  report to their offering.  , ,     (Logo: ... forecast the global multimodal biometrics market to grow ... 2016-2020.  Multimodal biometrics is being implemented ... healthcare, BFSI, transportation, automotive, and government for controlling ...
Breaking Biology News(10 mins):
(Date:5/23/2016)... ... , ... The need for blood donations in South Texas and across the nation is growing. ... Tissue Center, blood donations are on the decline. In fact, donations across the country are ... in South Texas in the last four years alone. , There is no substitute for ...
(Date:5/23/2016)... LONDON , May 23, 2016 ... Boost Efficiency by 40% - Frontage Implement a Single ... Enforce Quality, Compliance and Traceability Within the Bioanalytical lab ... the United States and China ... deployed across its laboratory facilities. In addition to serving as the ...
(Date:5/20/2016)... ... 2016 , ... The leading Regenerative Veterinary Medicine Company, VetStem Biopharma ., ... over 100 of their own patients with the VetStem Cell Therapy. Each of these ... for their patients. , The veterinarians are Dr Ross Rich former owner of ...
(Date:5/19/2016)... ... ... Anton Paar USA, located in Ashland, Virginia is pleased to announce that ... adds a third office building to the current facilities. , Growth is nothing ... along with office space adjacent to the previous main building. Through remodels and new ...
Breaking Biology Technology: