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:1/22/2016)... 22, 2016 ... of the  "Global Behavioral Biometric Market ... --> http://www.researchandmarkets.com/research/4lmf2s/global_behavioral ) has announced ... Biometric Market 2016-2020"  report to their ... Markets ( http://www.researchandmarkets.com/research/4lmf2s/global_behavioral ) has announced the ...
(Date:1/21/2016)... January 21, 2016 ... new market research report "Emotion Detection and Recognition Market by ... Tools (Facial Expression, Voice Recognition and Others), Services, ... forecast to 2020", published by MarketsandMarkets, the global ... reach USD 22.65 Billion by 2020, at a ...
(Date:1/20/2016)... Jan. 20, 2016   MedNet Solutions , an ... spectrum of clinical research, is pleased to announce the ... achievements are the result of the company,s laser focus ... eClinical , it,s comprehensive, easy-to-use and highly affordable ... --> Key MedNet growth achievements in 2015 include: ...
Breaking Biology News(10 mins):
(Date:2/8/2016)... active R&D program for the development of future natural products ... unique research and development center in Israel ... Stockton has a variety of products ... is active in more than 35 counties worldwide. ... flagship product Timorex Gold ® is used to ...
(Date:2/5/2016)... ATCC, the premier global biological materials resource ... and life science researchers that are working to address ... CDC website . --> CDC ... a single-stranded RNA virus of the Flaviviridae family, genus ... Chikungunya Viruses. Zika virus is transmitted to humans primarily ...
(Date:2/4/2016)... , ... February 04, 2016 , ... ... development and compliance training, today announced an interactive FDA compliance training ... The RAPS (Regulatory Affairs Professional Society) accredited interactive course on Morf Playbook—now conveniently ...
(Date:2/4/2016)... Beike Biotechnology, the Shenzhen ... ceremony in late 2015 to mark their successful combined ... --> --> The ... Cell Therapy" was hosted by the Shenzhen Cell Bank ... of Beike Biotechnology Co., Ltd. Shenzhen,s ...
Breaking Biology Technology: