Leiden University Scholarly Publications

Efficient and scalable generation of primordial germ cells in 2D culture using basement membrane extract overlay

Precise homology-directed installation of large genomic edits in human cells with cleaving and nicking high-specificity Cas9 variants

Large-scale genome editing based on high-capacity adenovectors and CRISPR-Cas9 nucleases rescues full-length dystrophin synthesis in DMD muscle cells

Broadening the reach and investigating the potential of prime editors through fully viral gene-deleted adenoviral vector delivery

TGF-beta-induced endothelial to mesenchymal transition is determined by a balance between SNAIL and ID factors

Precise and broad scope genome editing based on high-specificity Cas9 nickases

Novel therapeutic approaches for the treatment of retinal degenerative diseases: focus on CRISPR/Cas-based gene editing

A primer to gene therapy

High-capacity adenoviral vectors permit robust and versatile testing of DMD gene repair tools and strategies in human cells

Adenoviral vectors meet gene editing: a rising partnership for the genomic engineering of human stem cells and their progeny

Expanding the editable genome and CRISPR-Cas9 versatility using DNA cutting-free gene targeting based on in trans paired nicking

Integrating gene delivery and gene-editing technologies by adenoviral vector transfer of optimized CRISPR-Cas9 components

Intronic SMCHD1 variants in FSHD: testing the potential for CRISPR-Cas9 genome editing

The Chromatin Structure of CRISPR-Cas9 Target DNA Controls the Balance between Mutagenic and Homology-Directed Gene-Editing Events

DNA, RNA, and Protein Tools for Editing the Genetic Information in Human Cells

In trans paired nicking triggers seamless genome editing without double-stranded DNA cutting

The Chromatin Structure Differentially Impacts High-Specificity CRISPR-Cas9 Nuclease Strategies

Correction of Recessive Dystrophic Epidermolysis Bullosa by Transposon-Mediated Integration of COL7A1 in Transplantable Patient-Derived Primary Keratinocytes

Adenoviral vectors encoding CRISPR/Cas9 multiplexes rescue dystrophin synthesis in unselected populations of DMD muscle cells

Probing the impact of chromatin conformation on genome editing tools

The emerging role of viral vectors as vehicles for DMD gene editing

Engineered Viruses as Genome Editing Devices

Selection-free gene repair after adenoviral vector transduction of designer nucleases: rescue of dystrophin synthesis in DMD muscle cell populations

Genome editing at the crossroads of delivery, specificity, and fidelity