AAV2-mediated gene therapy for Bietti crystalline dystrophy provides functional CYP4V2 in multiple relevant cell models.
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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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The Scope of Pathogenic ABCA4 Mutations Targetable by CRISPR DNA Base Editing Systems—A Systematic Review
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Mirtron-mediated RNA knockdown/replacement therapy for the treatment of dominant retinitis pigmentosa
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In Silico Analysis of Pathogenic CRB1 Single Nucleotide Variants and Their Amenability to Base Editing as a Potential Lead for Therapeutic Intervention.
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CRISPR Systems Suitable for Single AAV Vector Delivery.
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Characterizing the cellular immune response to subretinal AAV gene therapy in the murine retina
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Therapy Approaches for Stargardt Disease
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Insights on the Regeneration Potential of Müller Glia in the Mammalian Retina
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Gene Therapy Rescues Cone and Rod Function in a Pre-Clinical Model of CDHR1-Associated Retinal Degeneration through Restoration of Photoreceptor Outer Segments
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Deep phenotyping of the Cdhr1-/- mouse validates its use in pre-clinical studies for human CDHR1-associated retinal degeneration
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Accurate Quantification of AAV Vector Genomes by Quantitative PCR
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Functional expression of complement factor I following AAV-mediated gene delivery in the retina of mice and human cells.
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AAV Induced Expression of Human Rod and Cone Opsin in Bipolar Cells of a Mouse Model of Retinal Degeneration
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Analysis of Pathogenic Variants Correctable With CRISPR Base Editing Among Patients With Recessive Inherited Retinal Degeneration.
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Is subretinal AAV gene replacement still the only viable treatment option for choroideremia?
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An analysis of the Kozak consensus in retinal genes and its relevance to gene therapy.
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CRISPR genome engineering for retinal diseases
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