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Levels Of Deficient Protein In Friedreich’s Ataxia boosted by New Gene Therapy Strategy

A novel approach to gene therapy that instructs a person’s own cells to produce more of a natural disease-fighting could offer a solution to treating many . 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. The article is available free online at the Human Gene Therapy website. *

The innovative gene therapy method described by Jacques Tremblay, , ZoĆ© Coulombe, and , Laval University, Quebec, and , 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. **

“This is a very clever approach to treat a recessive disease caused by decreased quantity of an otherwise normal protein,” says , 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.


* http://www.liebertpub.com/hum
Mary Ann Liebert, Inc./Genetic Engineering News