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Targeted Gene Silencing Drugs Are More Than 500 Times More Effective with New Delivery Method
New Rochelle, NY, December 20, 2012—Small interfering RNAs (siRNAs) are a potent new drug class that can silence a disease-causing gene, but delivering them to a target cell can be challenging. An innovative delivery approach that dramatically increases the efficacy of an siRNA drug targeted to the liver and has made it possible to test the drug in non-human primates is described in an article in Nucleic Acid Therapeutics, a peer-reviewed journal from Mary Ann Liebert, Inc. publishers. The article is available on the Nucleic Acid Therapeutics website.
In the article “Co-Injection of a Targeted, Reversibly Masked Endosomolytic Polymer Dramatically Improves the Efficacy of Cholesterol-Conjugated Small Interfering RNAs In Vivo” Wong and colleagues from Arrowhead Madison Inc. (Madison, WI) present a novel strategy to overcome the difficulty in delivering high levels of a gene knockdown siRNA drug to liver cells. While the cholesterol-conjugated siRNA drug is taken up preferentially by the liver, it is encapsulated in membrane-bound globules called endosomes and cannot reach the cells' DNA to exert its gene silencing effect. The researchers co-injected a polymer with the drug that also targets the liver and, once inside liver cells, breaks open the endosomes, releasing the siRNA drug.
“The promise of siRNAs is as strong as ever and is becoming even more so with progress in delivering these molecules to the right place at the right time," says Executive Editor Fintan Steele, PhD, SomaLogic, Inc., Boulder, CO. "The work by Wong and colleagues is another important step towards realizing this promise.”
Nucleic Acid Therapeutics is under the editorial leadership of Co-Editors-in-Chief Bruce A. Sullenger, PhD, Duke Translational Research Institute, DukeUniversity Medical Center, Durham, NC, and C.A. Stein, MD, PhD, City of Hope National Medical Center, Duarte, CA; and Executive Editor Fintan Steele, PhD, SomaLogic, Boulder, CO.
About the Journal
Nucleic Acid Therapeutics is an authoritative, peer-reviewed journal published bimonthly in print and online that focuses on cutting-edge basic research, therapeutic applications, and drug development using nucleic acids or related compounds to alter gene expression. Nucleic Acid Therapeutics is the Official Journal of the Oligonucleotide Therapeutics Society. Complete tables of content and a free sample issue may be viewed on the Nucleic Acid Therapeutics website.
About the Publisher
Mary Ann Liebert, Inc. publishers is a privately held, fully integrated media company known for establishing authoritative peer-reviewed journals in many promising areas of science and biomedical research, including Human Gene Therapy,, Genetic Testing and Molecular Biomarkers, ASSAY and Drug Development Technologies, and DNA and Cell Biology. Its biotechnology trade magazine, Genetic Engineering & Biotechnology News (GEN), was the first in its field and is today the industry’s most widely read publication worldwide. A complete list of the firm’s 70 journals, books, and newsmagazines is available on the Mary Ann Liebert, Inc. publishers website.