New options for targeting gene mutation in FA described in nucleic acid therapeutics


Credit: Mary Ann Liebert, Inc., publishers

New Rochelle, NY, March 7, 2018–Researchers have shown that a wide variety of synthetic antisense oligonucleotides with different chemical modifications can activate the frataxin gene, which contains a mutation that decreases its expression in the inherited neurologic disorder Friedreich's ataxia (FA). This new finding, which demonstrates a broad range of flexible options for identifying novel compounds capable of increasing frataxin protein expression and alleviating the effects of FA, is published 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 until April 7, 2018.

The article entitled "Activation of Frataxin Protein Expression by Antisense Oligonucleotides Targeting the Mutant Expanded Repeat" is coauthored by David Corey UT Southwestern Medical Center at Dallas, TX and coauthors from Ionis Pharmaceuticals (Carlsbad, CA), McGill University (Montreal, Canada), and University of Massachusetts (Worcester, MA). The researchers show that various nucleic acid compounds with a range of chemical modifications are able to bind to the abnormal GAA repeat sequences in the FA gene. They demonstrated this in multiple cell lines derived from FA patients who had varied numbers of GAA repeats, implying a strong foundation for future drug development.

"The resources and long-term commitment required to pursue these types of investigations underline the needs and benefits of academia-industry collaborations that are advancing the field," 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.

Research reported in this publication was supported by the National Institutes of Health under Award Number GM R35118103. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.


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 Co-Editors-in-Chief Bruce A. Sullenger, PhD, Duke Translational Research Institute, Duke University Medical Center and Annemieke Aartsma-Rus, PhD, Leiden University Medical Center, 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.

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

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