| Literature DB >> 32270790 |
Ewa Czuba-Wojnilowicz1, Serena Viventi, Sara E Howden, Simon Maksour, Amy E Hulme, Christina Cortez-Jugo, Mirella Dottori, Frank Caruso.
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.Entities:
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Year: 2020 PMID： 32270790 DOI： 10.1039/c9bm01757g
Source DB: PubMed Journal: Biomater Sci ISSN： 2047-4830 Impact factor: 6.843