Therapeutic RNAi-based gene therapy for neurodegenerative disorders : slowing down the ticking clock

Author(s):
Martier, R.M.
Published
2020
Publication type
Doctoral Thesis

Abstract

This thesis describes the development of microRNA-based gene therapies for ALS and

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.

ALSSCA3miRNAGene therapyC9orf72ATXN3AAVRNAi
Defense date
2020-05-27
Language
English
Supervisor(s)
Deventer, S.J. van
Advisor(s)
Konstantinova, P.
Committee member(s)
Roon-Mom, W.M.C. van; Maarel, S. van der; Hoeben, R.; Sampaio, C.; Nguyen, H.P.; Pasterkamp, R.J.
Qualification
Ph.D.
Degree granting institution
Faculty of Medicine, Leiden University Medical Center (LUMC), Leiden University, Leiden
hdl
1887/92292
ISBN(s)
9789461829870

Funding

Sponsorship
uniQure