Combinatorial testing of viral vector and CRISPR systems for precision genome editing

Author(s):
Li, Z.
Published
2025
Publication type
Doctoral Thesis

Abstract

The work present in this dissertation provides the evidence that delivery of well-established CRISPR/Cas9 gene editing machinery rationally allocated in different viral vectors can achieve plausibly ideal results of gene editing in the mammalian cells regarding efficiencies, accuracy and specificity. The use of CRISPR/Cas9 system makes the gene editing convenient and customized and compared to other delivery options, the viral vectors are outperformed due to natural evolution coffering high compatibility to mammalian cells. However, it would still be a long road to go along with all kinds of obstacles, then finally we may test in one clinical trial the concept of such combination investigated in the thesis.

Precision gene editingCRISPR/Cas9High-capacity adenoviral vectorAdeno-associated viral vectorHomology-directed repair
Defense date
2025-10-08
Language
English
Supervisor(s)
Hoeben, R.C.
Advisor(s)
Gonçalves, M.A.F.V.
Committee member(s)
Chuva de Sousa Lopes, S.M.; Pijnappel, W.W.M.; Heemskerk, M.H.M.; Dijke, P. ten; Lei, Z.Y.
Qualification
Ph.D.
Degree granting institution
Faculty of Medicine, Leiden University Medical Center (LUMC), Leiden University, Leiden
hdl
1887/4262567
ISBN(s)
9789465225685

Funding

Sponsorship
the China Scholarship Council (CSC)-Leiden University joint program; the Prinses Beatrix Spierfonds; the Duchenne Parent Project NL; the Dutch Research Council (NWO)-OpenTechnology Program; the EU Marie Skłodowska-Curie Doctoral Network Actions