Issue 9, 2020

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

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.

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

Supplementary files

Article information

Article type
Communication
Submitted
31 Oct 2019
Accepted
30 Mar 2020
First published
09 Apr 2020

Biomater. Sci., 2020,8, 2398-2403

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

E. Czuba-Wojnilowicz, S. Viventi, S. E. Howden, S. Maksour, A. E. Hulme, C. Cortez-Jugo, M. Dottori and F. Caruso, Biomater. Sci., 2020, 8, 2398 DOI: 10.1039/C9BM01757G

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