Drug delivery strategy eliminates myotonia symptoms in mice with myotonic dystrophy

©2013, Mary Ann Liebert, Inc., publishers

By 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.

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, , and , 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 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."

More information: The article is available free on the Nucleic Acid Therapeutics website.

add to favorites email to friend print save as pdf

Related Stories

Genetic source of muscular dystrophy neutralized

Jul 16, 2009

(PhysOrg.com) -- Researchers at the University of Rochester Medical Center have found a way to block the genetic flaw at the heart of a common form of muscular dystrophy. The results of the study, which were ...

Researchers reverse key symptom of muscular dystrophy

Nov 15, 2007

Researchers at the University of Rochester Medical Center have identified a compound that eliminates myotonia – a symptom of muscular dystrophy – in mice. The study was published online today in the Journal of Clinical In ...

Recommended for you

App for headache sufferers shows success

11 hours ago

A unique app that helps headache sufferers to record the severity and regularity of their pain is being used as part of a Griffith research study.

West African airline suspends flights amid Ebola (Update)

17 hours ago

(AP)—Police officers deployed to Liberia's international airport to ensure passengers are screened for Ebola symptoms as a major regional airline announced Tuesday it was suspending flights to the cities hardest hit by ...

User comments