PUBLIC RELEASE DATE:

17-Jul-2014

Contact: Kathryn Ruehle kruehle@liebertpub.com 914-740-2100 Mary Ann Liebert, Inc./Genetic Engineering News

Splice-Switching Oligonucleotide Therapeutics Is Promising New Method for Editing Gene Transcripts

New Rochelle, NY, July 17, 2014In splice-switching, an innovative therapeutic approach, targeted oligonucleotide drugs alter the editing of a gene transcript to produce the desired form of a protein. Developments in this rapidly advancing field have already led to promising treatments for such diseases as Duchenne Muscular Dystrophy and spinal muscular atrophy, as described in an article in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc., publishers. The article is available on the Human Gene Therapy website.

In "Development of Therapeutic Splice-Switching Oligonucleotides," Petra Disterer and coauthors from University College London, University of London, and Queen Mary University of London, UK, and Medical University of Warsaw, Poland, present an overview of the many possible therapeutic applications for splice-switching oligonucleotides. The authors discuss the design and chemical modification of these novel compounds to increase their stability and effectiveness, and emphasize the need to develop efficient solutions on a case by case basis.

"This is an emerging therapeutic area with promising clinical results," says James M. Wilson, MD, PhD, Editor-in-Chief of Human Gene Therapy, and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia.

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About the Journal

Human Gene Therapy, the official journal of the European Society of Gene and Cell Therapy, British Society for Gene and Cell Therapy, French Society of Cell and Gene Therapy, German Society of Gene Therapy, and five other gene therapy societies, is an authoritative peer-reviewed journal published monthly in print and online. Human Gene Therapy presents reports on the transfer and expression of genes in mammals, including humans. Related topics include improvements in vector development, delivery systems, and animal models, particularly in the areas of cancer, heart disease, viral disease, genetic disease, and neurological disease, as well as ethical, legal, and regulatory issues related to the gene transfer in humans. Its sister journals, Human Gene Therapy Methods, published bimonthly, focuses on the application of gene therapy to product testing and development, and Human Gene Therapy Clinical Development, published quarterly, features data relevant to the regulatory review and commercial development of cell and gene therapy products. Tables of content for all three publications and a free sample issue may be viewed on the Human Gene Therapy website.

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Splice-switching oligonucleotide therapeutics is new method for editing gene transcript