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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… Click to show full 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.
Keywords:
particle mediated;
mediated delivery;
friedreich ataxia;
frataxin;
neuronal model
Journal Title: Biomaterials science
Year Published: 2020
Link to full text (if available)
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Journal Title: Biomaterials science
Year Published: 2020
Link to full text (if available)
Share on Social Media: Sign Up to like & get
recommendations!