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Issue 9, 2020
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Particle-mediated delivery of frataxin plasmid to a human sensory neuronal model of Friedreich's ataxia

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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

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

Biomater. Sci., 2020,8, 2398-2403
Article type
Communication

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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