Therapeutic RNAi-based gene therapy for neurodegenerative disorders : slowing down the ticking clock
| Autor: | Martier, R.M. |
|---|---|
| Přispěvatelé: | Deventer, S.J. van, Konstantinova, P., Roon-Mom, W.M.C. van, Maarel, S. van der, Hoeben, R., Sampaio, C., Nguyen, H.P., Pasterkamp, R.J., Leiden University |
| Jazyk: | angličtina |
| Rok vydání: | 2020 |
| Předmět: | |
| Zdroj: | None |
| Popis: | This thesis describes the development of microRNA-based gene therapies for ALS andSCA3. Other aspects of a successful gene therapy including the administration routes toreach the target organs and regulation of the transgene expression were also investigated.Due to the permanent nature of gene therapy, a method to modulate the transgeneexpression is desirable. |
| Databáze: | OpenAIRE |
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