Venema, Rindert (2023) Delivery of therapeutic antisense oligonucleotides for exon skipping in epidermolysis bullosa. Master's Research Project 2, Biomedical Sciences.
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Abstract
We investigated and optimized delivery of a fluorescently labelled fab-conjugated antisense oligonucleotide (AON) construct (flAON-Fab conjugate) for exon skipping in dystrophic epidermolysis bullosa (DEB) to gain insights in its cellular uptake, distribution and effectiveness. Second, we sought the most potent AON sequence by optimizing transfection of naked AONs in primary keratinocytes, and subsequently performing an ‘Oligowalk’ in which we transfected 102 unique AON constructs, specifically targeting exon 105 of the COL7A1 gene. Lastly, we optimized a 3D skin model of human fibroblasts, keratinocytes and a collagen mix, to resemble the human skin as closely as possible that can potentially serve as a null-model or replacement model for human skin in EB.
| Item Type: | Thesis (Master's Research Project 2) |
|---|---|
| Supervisor name: | Laman, J.D. and Bremer, J. |
| Degree programme: | Biomedical Sciences |
| Thesis type: | Master's Research Project 2 |
| Language: | English |
| Date Deposited: | 20 Jul 2023 07:15 |
| Last Modified: | 20 Jul 2023 07:15 |
| URI: | https://fse.studenttheses.ub.rug.nl/id/eprint/30796 |
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