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

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.