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Development, characterization and synthesis of multi-specific proteins for targeted delivery of nucleic acids and nucleic acid derivatives

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Killian, Tobias Friedrich
Date of publication of this fulltext: 05 Aug 2019 13:56

Abstract (English)

The focus of this PhD thesis is the development of a novel strategy for specific and efficient delivery of gene expression systems for targeted genome editing. To mediate efficient transgene expression only inside the nuclei of the cells of interest, an optimization of every step along the gene delivery route as outlined in the following is absolutely mandatory: 1. Specific delivery to the ...


Translation of the abstract (German)

Die vorliegende Doktorarbeit befasst sich mit der Entwicklung eines Systems für die gezielte Verabreichung von Transgenen zur spezifischen, therapeutischen Genom-Editierung. Damit ein Gentherapeutikum systemisch optimal wirksam ist, müssen drei Schritte gezielt adressiert werden: 1. Spezifische Aufnahme des Transgens allein durch die Zielzelle 2. Effiziente Translokation des Transgens in den ...


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