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, Frank Caruso

Biomaterials science 2020 May 07

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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.

Citation

Ewa Czuba-Wojnilowicz, Serena Viventi, Sara E Howden, Simon Maksour, Amy E Hulme, Christina Cortez-Jugo, Mirella Dottori, Frank Caruso. Particle-mediated delivery of frataxin plasmid to a human sensory neuronal model of Friedreich's ataxia. Biomaterials science. 2020 May 07;8(9):2398-2403