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

Wednesday, July 25, 2012 - 11:33 in Biology & Nature

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.

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