Leiden University Scholarly Publications

Characterization and AAV-mediated CRB gene augmentation in human-derived CRB1KO and CRB1KOCRB2+/- retinal organoids

Tagged IDS causes efficient and engraftment- independent prevention of brain pathology during lentiviral gene therapy for Mucopolysaccharidosis type II

Human iPSC-derived preclinical models to identify toxicity of tumor-specific T cells with clinical potential

Evidence generation and reproducibility in cell and gene therapy research: a call to action

Correction of pathology in mice displaying Gaucher disease type 1 by a clinically-applicable lentiviral vector

AAV-CRB2 protects against vision loss in an inducible CRB1 retinitis pigmentosa mouse model

Cerebral organoids

Successful preclinical development of gene therapy for recombinase-activating gene-1-deficient SCID

Enhanced factor IX activity following administration of AAV5-R338L "Padua" factor IX versus AAV5 WT human factor IX in NHPs

AAV5-miHTT lowers huntingtin mRNA and protein without off-target effects in patient-derived neuronal cultures and astrocytes

Development of an AAV-based microRNA gene therapy to treat Machado-Joseph disease

AAV5-miHTT gene therapy demonstrates sustained Huntingtin lowering and functional improvement in Huntington disease mouse models

Integrated Human Evaluation of the Lysophosphatidic Acid Pathway as a Novel Therapeutic Target in Atherosclerosis

Clinical development of gene- and cell-based therapies: overview of the European landscape