Campagne, Lancewell (2020) HUNTINGTON’S DISEASE: A disease overview and future curative prospects through CRISPR/Cas9 genetic editing. Bachelor's Thesis, Biology.
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Abstract
Huntington's disease is a progressive and disastrous disease. Since the moment the gene causing HD was found in 1993, a lot has improved in our understanding of HD on a cellular and genetic level. To translate all relevant scientific understanding of HD into suitable patient specific models, will take a large amount of time and research. Removing a number of critical CAG repeats from the IT15 gene, or completely deactivating the IT15 gene using CRISPR/Cas9 are feasible therapeutic options for the future. Before human trials with CRISPR/Cas9 are possible, this too will need to be improved upon. There must be a reduction of off-target edits along with the prevention of deleterious CRISPR/Cas9-induced mutations. The current way of administering CRISPR/Cas9 involving viral delivery has shown to pose problems as well. Improving animal models and creating systems providing maximum safety are essential in supporting clinical trials in the future. Recent in vivo research using CRISPR/Cas9 has shown it might already be experimentally possible to remove the critical amount of CAG repeats in HD patients and ameliorate neurodegeneration (Stadtmauer et al., 2020). But for a therapeutic intervention to become widespread, the method has to be completely safe and all current technical obstacles have to be overcome.
| Item Type: | Thesis (Bachelor's Thesis) |
|---|---|
| Supervisor name: | Foijer, F. |
| Degree programme: | Biology |
| Thesis type: | Bachelor's Thesis |
| Language: | English |
| Date Deposited: | 25 Sep 2020 12:15 |
| Last Modified: | 28 Sep 2020 11:41 |
| URI: | https://fse.studenttheses.ub.rug.nl/id/eprint/23424 |
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