title: Tissue-Specific Promoters in the CNS.
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.
authors.affiliation: 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.
authors.forename: Sebastian
authors.initials: S
authors.lastname: Kügler
mesh_terms: D000818:Animals; D002490:Central Nervous System / Q000378:metabolism*; D000229:Dependovirus / Q000235:genetics*; D015870:Gene Expression; D015316:Genetic Therapy; D005822:Genetic Vectors / Q000008:administration & dosage; D006801:Humans; D009928:Organ Specificity; D011401:Promoter Regions, Genetic; D014158:Transcription, Genetic
keywords: AAV; Enhancer; Mir-binding site; Promoter; Transcription blocker; Transcriptional targeting; Vector tropism