New study shows a breadth of antisense drug activity across many different organs

December 10, 2013

New Rochelle, NY, December 10, 2013--Antisense therapeutics, a class of drugs comprised of short nucleic acid sequences, can target a dysfunctional gene and silence its activity. A new study has shown that antisense drugs delivered systemically show activity in a wide range of tissues and organs, supporting their broad therapeutic potential in many disease indications, as 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 Therapeuticswebsite.

Gene Hung, Xiaokun Xiao, Raechel Peralta, Gourab Bhattacharjee, Sue Murray, Dan Norris, Shuling Guo, and Brett Monia, Isis Pharmaceuticals, Carlsbad, CA, developers of antisense therapeutics, compared two antisense drug chemistries (Generation 2.0 and 2.5) designed to target a gene that is expressed by virtually all cells in mice and non-human primates. They demonstrated antisense activity in many tissues and cell types, including liver, kidney, lung, muscle, adipose, adrenal gland, and peripheral nerves. The Generation 2.5 antisense compound was more effective in a wider range of tissues, according to the results presented in the article "Characterization of Target mRNA Reduction Through In Situ RNA Hybridization in Multiple Organ Systems Following Systemic Antisense Treatment in Animals."

"This seminal work addresses one of the most important questions facing the field, the demonstration and evaluation of multiple organ targeting by Nucleic Acid Therapeutics," 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. "This publication provides a benchmark for convergent analyses in multiple models for preclinical efficacy evaluation."

Nucleic Acid Therapeutics is under the editorial leadership of Co-Editors-in-Chief Bruce A. Sullenger, PhD, Duke Translational Research Institute, Duke University Medical Center, Durham, NC, and C.A. Stein, MD, PhD, City of Hope National Medical Center, Duarte, CA; and Executive Editor Graham C. Parker, PhD.
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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 Therapeuticswebsite.

About the Society

The Oligonucleotide Therapeutics Society is an open, nonprofit 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 promising areas of science and biomedical research, including Human Gene Therapy, Human Gene Therapy Methods, Human Gene Therapy Clinical Development, 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 80 journals, books, and newsmagazines is available on the Mary Ann Liebert, Inc., publishers website.

Mary Ann Liebert, Inc. 140 Huguenot St., New Rochelle, NY 10801-5215 Phone: (914) 740-2100 (800) M-LIEBERT Fax: (914) 740-2101 http://www.liebertpub.com

Mary Ann Liebert, Inc./Genetic Engineering News

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