CORDIS Project
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This project focuses on improving gene therapy by enhancing the delivery of nucleic acids into cells. It aims to overcome barriers to DNA transport using small-molecule inhibitors and engineered nanoparticles, ultimately increasing the efficiency of gene transfection for therapeutic applications.
Safe and efficient delivery of nucleic acids to tissues and cells is a shared challenge in the clinical translation of gene therapy and gene editing.
At the intracellular level, DNA delivery is hindered by endo/lysosomal sequestration, inefficient transport into the nucleus and a retention mechanism mediated by the barrier-to-autointegration factor (BAF) protein that detects, clusters and locks away intruding double-stranded DNA in membrane cages.
The first two of these intracellular obstacles a…
EIDGENOESSISCHE TECHNISCHE HOCHSCHULE ZUERICH
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