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Tissue-Specific Promoters in the CNS

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Gene Therapy for Neurological Disorders

Part of the book series: Methods in Molecular Biology ((MIMB,volume 1382))

Abstract

This chapter outlines some general principles of transcriptional targeting approaches using viral vectors in the central nervous system. Transcriptional targeting is first discussed in the context of vector tropism and appropriate delivery. Then, some of our own attempts to restrict expression of therapeutic factors to distinct brain cell populations are discussed, followed by a detailed description of the setscrews that are available for these experiments. A critical discussion of current stumbling blocks and necessary developments to achieve clinical applicability of advanced targeted vector systems is provided.

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Correspondence to Sebastian Kügler Ph.D. .

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Kügler, S. (2016). Tissue-Specific Promoters in the CNS. 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_6

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  • DOI: https://doi.org/10.1007/978-1-4939-3271-9_6

  • Publisher Name: Humana Press, New York, NY

  • Print ISBN: 978-1-4939-3270-2

  • Online ISBN: 978-1-4939-3271-9

  • eBook Packages: Springer Protocols

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