Persistent URL of this record https://hdl.handle.net/1887/92292
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Therapeutic RNAi-based gene therapy for neurodegenerative disorders : slowing down the ticking clock
SCA3. Other aspects of a successful gene therapy including the administration routes to
reach 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 transgene
expression is desirable.
- All authors
- Martier, R.M.
- Supervisor
- Deventer, S.J. van
- Co-supervisor
- Konstantinova, P.
- Committee
- Roon-Mom, W.M.C. van; Maarel, S. van der; Hoeben, R.; Sampaio, C.; Nguyen, H.P.; Pasterkamp, R.J.
- Qualification
- Doctor (dr.)
- Awarding Institution
- Faculty of Medicine, Leiden University Medical Center (LUMC) , Leiden University
- Date
- 2020-05-27
- ISBN
- 9789461829870
Funding
- Sponsorship
- uniQure