Abstract
The use of antisense oligonucleotides to target specific mRNA sequences represents a promising therapeutic strategy for neurological disorders. Recent advances in antisense technology enclose the development of phosphorodiamidate morpholino oligomers (MO), which is one of the best candidates for molecular therapies due to MO’s excellent pharmacological profile.
Nevertheless, the route of administration of antisense compounds represents a critical issue in the neurological field. Particularly, as regards motor neuron diseases, intracerebroventricular (ICV) injection is undoubtedly the most efficient procedure to directly deliver therapeutic molecules in the central nervous system (CNS). Indeed, we recently demonstrated the outstanding efficacy of the MO antisense approach by its direct administration to CNS of the transgenic mouse models of Spinal Muscular Atrophy (SMA) and Amyotrophic Lateral Sclerosis (ALS).
Here, we describe methods to perform the ICV delivery of MO in neonatal SMA mice and in adult ALS mice.
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Acknowledgement
This work was supported by AriSLA, Pilot Grant ALSsiMO to M.N. and Telethon, GGP14025 to M.N.
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Nizzardo, M., Rizzuti, M. (2017). Intracerebroventricular Delivery in Mice for Motor Neuron Diseases. In: Moulton, H., Moulton, J. (eds) Morpholino Oligomers. Methods in Molecular Biology, vol 1565. Humana Press, New York, NY. https://doi.org/10.1007/978-1-4939-6817-6_19
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DOI: https://doi.org/10.1007/978-1-4939-6817-6_19
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