Novel CNS gene delivery systems

Abstract

Gene therapy for neurological disorders requires carriers for the therapeutic genes which can safely and efficiently carry the genes into desired cell types. The aim of this study was to exploit suitable gene carrier which has the potential for CNS gene delivery and to improve their performances in terms of efficiency, biosafety, and specificity. Both non-viral and viral vectors were involved in this research. For the non-viral vector part, a newly developed biodegradable polymer, PPE-EA, was adopted in CSF gene delivery. The results established the potential of PPE-EA as a new and biocompatible gene carrier to achieve sustained gene expression in CNS. For the viral vector part, a newly emerged viral gene delivery vector, baculovirus, was modified to achieve neuronal specific gene delivery by utilizing a hybrid neuronal specific promoter, CMV E/PDGF. The retrograde and anterograde axonal transport profile of this recombinant baculovirus was systemically studies in several CNS pathways.