A new gene therapy method achieved a 2- to 3-fold increase in production of a protein deficient in patients with Friedreich’s ataxia, according to an article published Instant Online in Human Gene Therapy, a peer-reviewed journal from GEN parent company Mary Ann Liebert, Inc.
The article, “TALE Proteins Induce the Expression of the Frataxin Gene", is available free online at the Human Gene Therapy website.
Co-authors Jacques Tremblay, Pierre Chapdelaine, Zoé Coulombe, and Joel Rousseau—affiliated with Quebec’s Laval University and the University of Quebec—detail the gene therapy method, which instructs a person’s own cells to produce more of a natural disease-fighting protein that could treat several genetic disorders.
The method uses the ability of a family of proteins called Tal effector (TALE) proteins to target specific DNA sequences. The authors engineered TALE proteins to target the gene that codes for the frataxin protein, which is deficient in Friedreich’s ataxia, as a model of how this method could be used to treat genetic disease.
The ability to induce cells to produce more frataxin could reduce symptoms of the disease and provide an effective, long-term therapeutic strategy, the authors conclude.
“This is a very clever approach to treat a recessive disease caused by decreased quantity of an otherwise normal protein,” says James M. Wilson, M.D., Ph.D., editor-in-chief, and director of the Gene Therapy Program, Department of Pathology and Laboratory Medicine, University of Pennsylvania Perelman School of Medicine, Philadelphia.