New gene therapy strategy boosts levels of deficient protein in Friedreich's ataxia

The method was used to achieve a 2- to 3-fold increase in production of a protein deficient in patients with Friedreich's ataxia, as described in an article published Instant Online in Human Gene Therapy, a peer-reviewed journal from Mary Ann Liebert, Inc. (http://www.liebertpub.com) The article is available free online at the Human Gene Therapy website (http://www.liebertpub.com/hum).

The innovative gene therapy method described by Jacques Tremblay, Pierre Chapdelaine, Zoé Coulombe, and Joel Rousseau, Laval University, Quebec, and University of Quebec, Canada, takes advantage of the ability of a family of proteins called Tal effector (TALE) proteins to target specific DNA sequences. As a model of how this method could be used to treat genetic disease, the authors engineered TALE proteins to target the gene that codes for the frataxin protein, which is deficient in Friedreich's ataxia.

The ability to induce cells to produce more frataxin could reduce symptoms of the disease and provide an effective, long-term therapeutic strategy, conclude the authors in the article “TALE Proteins Induce the Expression of the Frataxin Gene. (http://online.liebertpub.com/doi/full/10.1089/hum.2012.034)

“This is a very clever approach to treat a recessive disease caused by decreased quantity of an otherwise normal protein,” says James M. Wilson, MD, PhD, Editor-in-Chief, and Director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia.

About the Journal
Human Gene Therapy (http://www.liebertpub.com/hum), 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 that 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. Tables of content and a free sample issue may be viewed online at the Human Gene Therapy website (http://www.liebertpub.com/hum).

About the Publisher

Mary Ann Liebert, Inc., publishers (http://www.liebertpub.com) 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 Tissue Engineering, Stem Cells and Development, and Cellular Reprogramming. 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 at Mary Ann Liebert, Inc. (http://www.liebertpub.com)

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