Journal article

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 | Royal Society of Chemistry | Published : 2020

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.

Grants

Awarded by Australian Research Council Centre of Excellence in Convergent Bio-Nano Science and Technology


Awarded by National Health and Medical Research Council Senior Principal Research Fellowship


Funding Acknowledgements

This research was conducted and funded by the Australian Research Council Centre of Excellence in Convergent Bio-Nano Science and Technology (project number CE140100036). F. C. acknowledges the award of a National Health and Medical Research Council Senior Principal Research Fellowship (GNT1135806). S. V. was funded by a Melbourne International Research Scholarship and a Melbourne International Fee Remission Scholarship (The University of Melbourne). This work was performed in part at the Materials Characterisation and Fabrication Platform (MCFP) at The University of Melbourne and the Victorian Node of the Australian National Fabrication Facility (ANFF). This study was also supported by funding from the National Ataxia Foundation, Friedreich's Ataxia Research Alliance USA, Friedreich's Ataxia Research Association Australasia, The University of Melbourne and University of Wollongong. We thank Dr Gyeongwon Yun and Zhixing Lin for their assistance with AFM and SEM imaging, respectively. All experiments were performed in accordance with the Guidelines of the National Health and Medical Research Council, and experiments were approved by the ethics committee at The University of Melbourne (1545394 and 0829937). Informed consents were obtained from human participants of this study.