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Psychometric Validation of the ViSIO-PRO and ViSIO-ObsRO in Retinitis Pigmentosa and Leber Congenital Amaurosis.
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Extra-viral DNA in adeno-associated viral vector preparations induces TLR9-dependent innate immune responses in human plasmacytoid dendritic cells.
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Trendbericht Anorganische Chemie 2023: Hauptgruppen
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Gene therapy for inherited retinal disease: long-term durability of effect.
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Plasmid-mediated gene transfer of Cas9 induces vector-related but not SpCas9-related immune responses in human retinal pigment epithelial cells.
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Development of a functional assay for the assessment of two common CRB1 mutations
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Generation of qualitative evidence to support the content validity of the ViSIO-PRO and ViSIO-ObsRO in Retinitis Pigmentosa and Leber Congenital Amaurosis
Conference paper
Kay CN. et al, (2022), INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 63
Neural plasticity of the human visual system reflects treatment effect in gene therapy for Achromatopsia
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PERCEIVE study report: Real-world safety and effectiveness of voretigene neparvovec
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Psychometric validation of patient-reported outcome (PRO) and observer-reported outcome (ObsRO) measures in Retinitis Pigmentosa and Leber Congenital Amaurosis
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Audo IS. et al, (2022), INVESTIGATIVE OPHTHALMOLOGY & VISUAL SCIENCE, 63
Spatial and temporal resolution of the photoreceptors rescue dynamics after treatment with voretigene neparvovec.
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Development of retinal atrophy after subretinal gene therapy with voretigene neparvovec.
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Envisioning the development of a CRISPR-Cas mediated base editing strategy for a patient with a novel pathogenic CRB1 single nucleotide variant.
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Central Visual Function and Genotype-Phenotype Correlations in PDE6A-Associated Retinitis Pigmentosa.
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Early disruption of photoreceptor cell architecture and loss of vision in a humanized pig model of usher syndromes.
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Kinetics of Heterogeneous Background in Stargardt's Disease over Time.
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[From Bench to Bedside: Challenges in the Development of Genetherapeutic Approaches].
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Priglinger C. and Fischer MD., (2022), Klin Monbl Augenheilkd, 239, 261 - 262
Benefits and Shortcomings of Laboratory Model Systems in the Development of Genetic Therapies.
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Bucher K. et al, (2022), Klin Monbl Augenheilkd, 239, 263 - 269
Challenges to Gene Editing Approaches in the Retina.
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