More potent, inexpensive gene silencing agents described in Nucleic Acid Therapeutics


New Rochelle, NY, May 12, 2016–Combining the therapeutic potential and advantages of existing oligonucleotide-based approaches to turn off disease-related genes, a type of single-stranded silencing RNAs (ss-siRNAs) has shown significantly improved potency and activity. The chemical modification used to create these novel ss-siRNAs is both inexpensive and readily available to researchers, as described in an article in Nucleic Acid Therapeutics, a peer-reviewed journal from Mary Ann Liebert, Inc. publishers. The article is available free for download on the Nucleic Acid Therapeutics website until June 12, 2016.

In "Single-Stranded Silencing RNAs (ss-siRNAs): Hit Rate and Chemical Modification," Hannah Pendergraff, Alexandre Debacker, and Jonathan Watts, University of Southampton, U.K., and UMass Medical School, Worcester, present the key features of ss-siRNAs. These compounds combine the advantages of the two established mechanisms of gene silencing being used in nucleic acid-based drug development: single-stranded antisense oligonucleotides, and duplex RNA interference (siRNA). The researchers' attempts to develop and optimize ss-siRNAs based on chemical modification of active siRNA duplexes led to some compounds that lost their gene silencing activity and some with increased toxicity. One modification, however, led to increased silencing activity without affecting toxicity.

"Scientific research is resource intensive, and the advances described here help bring within reach tools to allow more researchers to ask and answer therapeutic questions and democratize the research endeavor," says Executive Editor Graham C. Parker, PhD, The Carman and Ann Adams Department of Pediatrics, Wayne State University School of Medicine, Children's Hospital of Michigan, Detroit, MI.


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. The Journal is under the editorial leadership of Editor-in-Chief Bruce A. Sullenger, PhD, Duke Translational Research Institute, Duke University Medical Center, Durham, NC, and Executive Editor Graham C. Parker, PhD. Nucleic Acid Therapeutics is the official journal of the Oligonucleotide Therapeutics Society. Complete tables of content and a sample issue may be viewed on the Nucleic Acid Therapeutics website.

About the Society

The Oligonucleotide Therapeutics Society is an open, non-profit forum to foster academia- and industry-based research and development of oligonucleotide therapeutics. The society brings together the expertise from different angles of oligonucleotide research to create synergies and to bring the field of oligonucleotides to its full therapeutic potential.

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, Assay and Drug Development Technologies, Applied In Vitro Toxicology, and DNA and Cell Biology. Its biotechnology trade magazine, GEN (Genetic Engineering & Biotechnology News), was the first in its field and is today the industry's most widely read publication worldwide. A complete list of the firm's 80 journals, books, and newsmagazines is available on the Mary Ann Liebert, Inc., publishers website.

Media Contact

Kathryn Ryan
[email protected]


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