Projektbeschreibung
Molekulares Gerüst für In-vivo-Gentherapieanwendungen
Bei den meisten In-vivo-Gentherapieanwendungen werden virale Vektoren eingesetzt, mit denen die gewünschten Gene effizient in die Zielzellen eingeschleust werden können, indem sie den viralen Infektionsprozess nachahmen. Der virale Gentransfer leidet jedoch unter Sicherheitsproblemen, wie z. B. der Insertionsmutagenese, und erfordert alternative, nicht virale Ansätze für den Gentransfer in Zellen. Das EU-finanzierte Projekt ENDOSCAPE schlägt vor, Moleküle einzusetzen, die das Entkommen aus Endosomen, den Zellorganellen, die für das Einfangen und den Abbau von internalisierten Molekülen verantwortlich sind, erleichtern. Die Idee besteht darin, ein molekulares Gerüst zu erstellen, das diese Enhancer zusammen mit dem gewünschten Gen und einem zielgerichteten Liganden umfasst, um das Problem der zytosolischen und nukleären Übertragung von Gentherapeutika zu lösen.
Ziel
Gene therapy is one of the most promising treatment options for future advanced therapies in a broad range of diseases. Successful gene delivery requires the recognition of target cells as well as cytosolic and nucleosolic uptake of the gene. Currently, non-viral based gene delivery such as transfection reagents are only suitable for in vitro applications, and clinical gene therapeutics delivery is accomplished via viral vectors, which still has major safety concerns as well as complex and costly manufacturing procedures, preventing future implementation for the treatment of diseases with large patients groups.
In the last 15 years, a class of secondary plant metabolites has been discovered that selectively mediates endosomal escape and cytoplasmic delivery of macromolecules specifically at low endosomal pH, thereby inducing a 40-fold enhanced gene delivery efficacy in vivo. However, endosomal escape enhancers and gene therapeutic product must be applied as two components, thus complicating therapeutic approval and clinical applicability.
The ENDOSCAPE technology platform will develop and collect proof of concept for a non-viral gene delivery technology where endosomal escape enhancers, gene therapeutic product and targeting ligand are all bound to one molecular scaffold. Proof of concept of the ENDOSCAPE technology has a major impact on the therapeutic opportunities for current and future macromolecule drugs for a broad range of diseases and large patient groups.
All this induces new biotech-based businesses, new research projects and creates new technology platforms for development of new macromolecule therapeutics for a broad range of disease indications. The non-viral based ENDOSCAPE technology will enhance therapeutic efficacy with lower therapeutic dose thereby reducing costs of healthcare, improving the health of patients worldwide, and strengthening the competitive landscape of the EU in the worldwide quest for such an advanced technology.
Wissenschaftliches Gebiet
Schlüsselbegriffe
Programm/Programme
Aufforderung zur Vorschlagseinreichung
Andere Projekte für diesen Aufruf anzeigenUnterauftrag
H2020-SC1-2018-Single-Stage-RTD
Finanzierungsplan
RIA - Research and Innovation actionKoordinator
10117 Berlin
Deutschland