Abstract
One of the challenges currently facing neuroscientists is the development of effective therapies based on the advances achieved on basic research. The use of genes as pro-drugs can be faced as an approach to reduce this gap. Furthermore, gene downregulation through the use of antisense strategies, including the recent introduction of RNA interference, is yet another tool with great therapeutic potential.
While viruses have proved to be the most efficient system to mediate the delivery of nucleic acids (mostly DNA), their use in a clinic scenario raises various safety concerns. Additionally, the regional specialization of the nervous system function dictates that a therapeutic intervention may be best achieved by the local and specific delivery of a therapeutic agent. In that sense a targeted delivery may avoid unwanted adverse effects that could result from a more ubiquitous delivery. In this chapter, the alternative strategies being explored to the delivery of nucleic acids (DNA, antisense and siRNA oligonucleotides) to the nervous system will be discussed, with particular focus on biomaterial-based systems.
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Acknowledgements
The authors would like to acknowledge the FEDER funds through the Programa Operacional Factores de Competitividade—COMPETE and the Portuguese funds through FCT—Fundação para a Ciência e a Tecnologia (contracts PTDC/CTM-NAN/115124/2009, HMSP-ICT/0020/2010 and PEst-C/SAU/LA0002/2011) that supported this work. PM is supported by a FCT post-doctoral fellowship (SFRH/BPD/76936/2011).
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Pêgo, A.P., Oliveira, H., Moreno, P.M. (2013). Biomaterial-Based Vectors for Targeted Delivery of Nucleic Acids to the Nervous System. In: Coelho, J. (eds) Drug Delivery Systems: Advanced Technologies Potentially Applicable in Personalised Treatment. Advances in Predictive, Preventive and Personalised Medicine, vol 4. Springer, Dordrecht. https://doi.org/10.1007/978-94-007-6010-3_7
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