Biomaterial-Based Vectors for Targeted Delivery of Nucleic Acids to the Nervous System
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 downregulati
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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. Keywords Gene therapy • Nanomedicine • Neurodegenerative diseases • Neuropathies • Non-viral vectors • Nucleic acid based therapeutics • DNA • siRNA • Antisense oligonucleotides • Personalized medicine
A.P. Pêgo () • P.M. Moreno INEB – Instituto de Engenharia Biomédica, Universidade do Porto, Rua do Campo Alegre, 823, 4150-180 Porto, Portugal e-mail: [email protected] H. Oliveira CNRS, Laboratoire de Chimie des Polymères Organiques (UMR5629), Université de Bordeaux/IPB, ENSCBP, 16 Avenue Pey Berland, 33607 Pessac Cedex, France J. Coelho (ed.), Drug Delivery Systems: Advanced Technologies Potentially Applicable in Personalised Treatment, Advances in Predictive, Preventive and Personalised Medicine 4, DOI 10.1007/978-94-007-6010-3__7, © Springer ScienceCBusiness Media Dordrecht 2013
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1 Introduction The nervous system is constituted of a network of nerve cells that by transmitting signals between different body parts are responsible for the orchestration of the body actions. In humans the nervous system can be divided in the central nervous system (CNS) that includes the brain, the spinal cord and the retina, and the peripheral nervous system (PNS) constituted by sensory and motor neurons outside the CNS. Sensory nerves, originated from clusters of neurons called ganglia, conduct sensory signals from organs and skin to the CNS. Motor nerves, originated from motor neurons situated in the spinal cord, transmit stimulatory signals from the CNS towards muscles and glands. Due to its crucial role, the impact of disease or a physical injury on the nervous system can have devastating consequences to the individual and very frequently significant societal and economical repercussions. Nervous system problems are common and encompass a large spectrum of traumatic injuries, diseases, tumors or iatrogenic lesions. Depending on the extent of the disorder it may result in a partial or tot
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