Particle-mediated delivery of frataxin plasmid to a human sensory neuronal model of Friedreich's ataxia

Increasing frataxin protein levels through gene therapy is envisaged to improve therapeutic outcomes for patients with Friedreich's ataxia (FRDA). A non-viral strategy that uses submicrometer-sized multilayered particles to deliver frataxin-encoding plasmid DNA affords up to 27 000-fold increase in frataxin gene expression within 2 days in vitro in a stem cell-derived neuronal model of FRDA. Multilayered particles in gene therapy for Friedreich's ataxia induce a 27 000-fold increase in frataxin gene expression in a patient-derived cell model.