Abstract
Gene therapy is a powerful tool for treating diseases, including neurological disorder such at amyotrophic lateral sclerosis. When delivered to the CNS, gene therapy vectors can provide prosurvival signals to neurons, knock down the expression of toxic proteins, or restore lost function. How to best deliver this type of therapeutic depends on the nature of the disease and the expected function of the transgene. Here we describe a method for parenchymal injection into rodent models, allowing for localized delivery of gene therapy vectors and other therapeutic molecules. This technique has been a robust mechanism for proof-of-principle experiments.
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McEachin, Z.T., Donsante, A., Boulis, N. (2016). Gene Therapy for the Treatment of Neurological Disorders: Amyotrophic Lateral Sclerosis. In: Manfredsson, F. (eds) Gene Therapy for Neurological Disorders. Methods in Molecular Biology, vol 1382. Humana Press, New York, NY. https://doi.org/10.1007/978-1-4939-3271-9_28
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DOI: https://doi.org/10.1007/978-1-4939-3271-9_28
Publisher Name: Humana Press, New York, NY
Print ISBN: 978-1-4939-3270-2
Online ISBN: 978-1-4939-3271-9
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