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

Date:
July 25, 2012
Source:
Mary Ann Liebert, Inc., Publishers
Summary:
A novel approach to gene therapy that instructs a person’s own cells to produce more of a natural disease-fighting protein could offer a solution to treating many genetic disorders. The method was used to achieve a 2- to 3-fold increase in production of a protein deficient in patients with Friedreich’s ataxia.

A novel approach to gene therapy that instructs a person's own cells to produce more of a natural disease-fighting protein could offer a solution to treating many genetic disorders. 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 in Human Gene Therapy.

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.

"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.


Story Source:

The above story is based on materials provided by Mary Ann Liebert, Inc., Publishers. Note: Materials may be edited for content and length.


Journal Reference:

  1. Jacques P. Tremblay, Pierre Chapdelaine, Zoé Coulombe, Joel Rousseau. Transcription Activator-Like Effector Proteins Induce the Expression of the Frataxin Gene. Human Gene Therapy, 2012; 120720084321004 DOI: 10.1089/hum.2012.034

Cite This Page:

Mary Ann Liebert, Inc., Publishers. "New gene therapy strategy boosts levels of deficient protein in Friedreich’s ataxia." ScienceDaily. ScienceDaily, 25 July 2012. <www.sciencedaily.com/releases/2012/07/120725105212.htm>.
Mary Ann Liebert, Inc., Publishers. (2012, July 25). New gene therapy strategy boosts levels of deficient protein in Friedreich’s ataxia. ScienceDaily. Retrieved July 25, 2014 from www.sciencedaily.com/releases/2012/07/120725105212.htm
Mary Ann Liebert, Inc., Publishers. "New gene therapy strategy boosts levels of deficient protein in Friedreich’s ataxia." ScienceDaily. www.sciencedaily.com/releases/2012/07/120725105212.htm (accessed July 25, 2014).

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