Lentiviral vector-mediated RNA silencing in the CNS

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25 Citations (Scopus)


RNA silencing is an established method for investigating gene function and has attracted particular interest due to the potential for generating RNA-based therapeutics. Utilising lentiviral vectors as an efficient delivery system that offers stable, long term expression in post-mitotic cells further enhances the therapeutical applicability of an RNA-based gene therapy for the CNS. In this review we provide an overview of both lentiviral vectors and RNA silencing as well as presenting design considerations for generating lentiviral vectors capable of RNA silencing. We go on to describe the current pre-clinical data regarding lentiviral vector mediated RNA silencing for CNS disorders and discuss the concerns of non-specific side-effects associated with lentiviral vectors and siRNAs and how these might be mitigated.
Original languageEnglish
Number of pages40
JournalHuman gene therapy methods
Publication statusE-pub ahead of print - 2013


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