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

Ewa Czuba-Wojnilowicz, Serena Viventi, Sara E. Howden, Simon Maksour, Amy E. Hulme, Christina Cortez-Jugo, Mirella Dottori and Frank Caruso; Biomater. Sci., 2020, Advance Article doi:10.1039/C9BM01757G

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.

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