CRISPR-Cas9 gene correction is a therapeutic tool which may abolish half of the known genetic disorders. To achieve this we need to deliver three macromolecules into the nucleus simultaneously, as well as trigger the homology-directed repair (HDR) pathway. The focus of this poster is on the role of mitosis on the state of these barriers, which in theory removes the nuclear membrane barrier and primes the cell for the HDR pathway.
The mitotic state of the cell skews the gene-editing outcome greatly, indicating a need to target mitotic cells for therapeutic gene correction.
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Danny Wilbie
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Utrecht University
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PhD Candidate
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Protein delivery, CRISPR, gene therapy
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I am a second year PhD candidate in the Department of Pharmaceutics at UU university, in the group of Enrico Mastrobattista. My research focusses on the feasibility of applying CRISPR/Cas gene editing for gene correction in vivo.
Shuxuan Zheng
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Heleen van der Veen
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Utrecht University
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Dirk Hondmann
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Biopharma research
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Maria Zwartkruis
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UMC Utrecht
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Vincent Portegijs
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UU, Faculty of Science
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UU, Faculty of Science
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PhD student
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neuroscience
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PhD student
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Bram Klein Wentink
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UU, Faculty of Science
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UU, Faculty of Science
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Tamanna Lashkari
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Utrecht University
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