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Methods Mol Biol. 2016;1382:81-91. doi: 10.1007/978-1-4939-3271-9_6.

Tissue-Specific Promoters in the CNS.

Author information

1
Department of Neurology, Center Nanoscale Microscopy and Physiology of the Brain (CNMPB), University Medicine Göttingen, Waldweg 33, 37073, Göttingen, Germany. sebastian.kuegler@med.uni-goettingen.de.

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.

KEYWORDS:

AAV; Enhancer; Mir-binding site; Promoter; Transcription blocker; Transcriptional targeting; Vector tropism

PMID:
26611580
DOI:
10.1007/978-1-4939-3271-9_6
[Indexed for MEDLINE]

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