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:2/6/2017)... Feb. 6, 2017 According to Acuity ... driving border authorities to continue to embrace biometric ... are 2143 Automated Border Control (ABC) eGates and ... at more than 163 ports of entry across ... 2016 achieving a combined CAGR of 37%. APC ...
(Date:2/2/2017)...  Central to its deep commitment to honor ... Japan Prize Foundation today announced the laureates of ... envelope in their respective fields of Life Sciences ... being recognized with the 2017 Japan Prize for ... to the advancement of science and technology, but ...
(Date:1/26/2017)... -- Acuity Market Intelligence today released the 2017 "Ten ... characterizes 2017 as a "breakout" year for biometrics ... new understanding of the potential benefits these technologies ... are often perceived as threats to privacy and ... Acuity Market intelligence. "However, taken together these technologies ...
Breaking Biology News(10 mins):
(Date:2/23/2017)... , ... February 23, 2017 ... ... announced today that in a published evaluation of multiple immunoassay-based threat detection ... Department of Energy Laboratory, PathSensors’ CANARY® biosensor threat detection technology was found ...
(Date:2/23/2017)... ... February 23, 2017 , ... Today, ... cortisol, CRP, adiponectin, uric acid, and/or other biomarkers or SNPs of interest) using ... Salimetrics’ SalivaLab , the relationship between insulin and other relevant biomarkers can ...
(Date:2/23/2017)... LOS ANGELES , Feb. 23, 2017  Capricor Therapeutics, ... cardiac and other medical conditions, today announced that Linda Marbán, ... at two upcoming investor conferences: Cowen ... 2017 at 10:00 am ET Boston, MA ... 14, 2017 at 9:00 am PT (12:00 pm ET) ...
(Date:2/23/2017)... and SAN FRANCISCO , ... privately-held regenerative medicine company, and Beyond Type 1, a ... type 1 diabetes, today announced a grant from Beyond ... functional cure for type 1 and other insulin-requiring diabetes.  ... has been developing innovative stem cell-derived cell replacement therapies ...
Breaking Biology Technology: