About Careers MedBlog Contact us
Medindia LOGIN REGISTER
Advertisement

Myotonia Symptoms in Mice With Myotonic Dystrophy Eliminated by Drug Delivery Strategy

by Kathy Jones on February 24, 2013 at 11:22 AM
Font : A-A+

Myotonia Symptoms in Mice With Myotonic Dystrophy Eliminated by Drug Delivery Strategy

Researchers have been focusing on ways to minimize some of the disabling symptoms of myotonic dystrophy since a long time. New research might have found a way to do so.

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.

Advertisement

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.

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.
Advertisement

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."



Source: Eurekalert
Advertisement

Advertisement
Advertisement

Recommended Reading

Latest Drug News

 FDA Issues Warning About Compounded Diabetes Drug Semaglutide Products
The FDA advises consumers not to use compounded medications as an alternative to known diabetic and weight reduction medications Ozempic, Rybelsus, and Wegovy.
 Parkinson's Unmet Needs Creates Path for New Entrants into the Market
Addressing the unmet needs of Parkinson's Disease by providing disease-modifying therapies could bring about a major shift in the way that patients are treated.
How Microrobots Could Help Treat Bladder Diseases?
Microrobots could swirl through a person's blood stream, search for targeted areas to treat for various ailments.
How Can Multivitamin Supplements Slow Cognitive Aging?
Supplementation with multivitamins is an inexpensive way for older adults to slow down memory loss.
 Ivosidenib Approved for Acute Myeloid Leukemia & Advanced Cholangiocarcinoma
Some people with an aggressive blood cancer called acute myeloid leukemia (AML) may soon have a new drug option called Ivosidenib that blocks the activity of IDH1 gene.
View All
This site uses cookies to deliver our services.By using our site, you acknowledge that you have read and understand our Cookie Policy, Privacy Policy, and our Terms of Use  Ok, Got it. Close
×

Myotonia Symptoms in Mice With Myotonic Dystrophy Eliminated by Drug Delivery Strategy Personalised Printable Document (PDF)

Please complete this form and we'll send you a personalised information that is requested

You may use this for your own reference or forward it to your friends.

Please use the information prudently. If you are not a medical doctor please remember to consult your healthcare provider as this information is not a substitute for professional advice.

Name *

Email Address *

Country *

Areas of Interests