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A compact unc45b-promoter drives muscle-specific expression in zebrafish and mouse

Rudeck, S.; Etard, C. 1; Khan, M. M.; Rottbauer, W.; Rudolf, R. 1; Strähle, U. 1; Just, S.
1 Institut für Toxikologie und Genetik (ITG), Karlsruher Institut für Technologie (KIT)

Abstract:

Summary: Gene therapeutic approaches to cure genetic diseases require tools to express the rescuing gene exclusively within the affected tissues. Viruses are often chosen as gene transfer vehicles but they have limited capacity for genetic information to be carried and transduced. In addition, to avoid off-target effects the therapeutic gene should be driven by a tissue-specific promoter in order to ensure expression in the target organs, tissues, or cell populations. The larger the promoter, the less space will be left for the respective gene. Thus, there is a need for small but tissue-specific promoters. Here, we describe a compact unc45b promoter fragment of 195 bp that retains the ability to drive gene expression exclusively in skeletal and cardiac muscle in zebrafish and mouse. Remarkably, the described unc45b promoter fragment not only drives muscle-specific expression but presents heat-shock inducibility, allowing a temporal and spatial quantity control of (trans)gene expression. Here, we demonstrate that the transgenic expression of the smyd1b gene driven by the unc45b promoter fragment is able to rescue the embryonically lethal heart and skeletal muscle defects in smyd1b-deficient flatline mutant zebrafish. ... mehr


Verlagsausgabe §
DOI: 10.5445/IR/1000058945
Veröffentlicht am 07.01.2022
Originalveröffentlichung
DOI: 10.1002/dvg.22953
Scopus
Zitationen: 4
Web of Science
Zitationen: 4
Dimensions
Zitationen: 4
Cover der Publikation
Zugehörige Institution(en) am KIT Institut für Toxikologie und Genetik (ITG)
Publikationstyp Zeitschriftenaufsatz
Publikationsjahr 2016
Sprache Englisch
Identifikator ISSN: 0192-253X, 1520-6408, 1526-954X, 1526-968X
KITopen-ID: 1000058945
HGF-Programm 47.01.01 (POF III, LK 01) Biol.Netzwerke u.Synth.Regulat. ITG+ITC
Erschienen in Genesis
Verlag John Wiley and Sons
Band 54
Heft 8
Seiten 431–438
Vorab online veröffentlicht am 13.06.2016
Nachgewiesen in Scopus
Dimensions
Web of Science
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