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

Journal Article


Abstract


  • 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.

Publication Date


  • 2020

Citation


  • Czuba-Wojnilowicz, E., Viventi, S., Howden, S. E., Maksour, S., Hulme, A. E., Cortez-Jugo, C., . . . Caruso, F. (2020). Particle-mediated delivery of frataxin plasmid to a human sensory neuronal model of Friedreich's ataxia. Biomaterials Science, 8(9), 2398-2403. doi:10.1039/c9bm01757g

Scopus Eid


  • 2-s2.0-85084272358

Start Page


  • 2398

End Page


  • 2403

Volume


  • 8

Issue


  • 9

Abstract


  • 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.

Publication Date


  • 2020

Citation


  • Czuba-Wojnilowicz, E., Viventi, S., Howden, S. E., Maksour, S., Hulme, A. E., Cortez-Jugo, C., . . . Caruso, F. (2020). Particle-mediated delivery of frataxin plasmid to a human sensory neuronal model of Friedreich's ataxia. Biomaterials Science, 8(9), 2398-2403. doi:10.1039/c9bm01757g

Scopus Eid


  • 2-s2.0-85084272358

Start Page


  • 2398

End Page


  • 2403

Volume


  • 8

Issue


  • 9