Gene Therapies in Clinical Development to Treat Retinal Disorders
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A hypomorphic variant of choroideremia is associated with a novel intronic mutation that leads to exon skipping
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Gene Therapy Trial on X-Linked Retinitis Pigmentosa Caused by Mutations in RPGR
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Is RPGR-related retinal dystrophy associated with systemic disease? A case series
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Outcomes and Adverse Effects of Voretigene Neparvovec Treatment for Biallelic RPE65-Mediated Inherited Retinal Dystrophies in a Cohort of Patients from a Single Center
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Developmental Expression of the Cell Cycle Regulator p16INK4a in Retinal Glial Cells: A Novel Marker for Immature Ocular Astrocytes?
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Changes in Retinal Sensitivity Associated With Cotoretigene Toliparvovec in X-Linked Retinitis Pigmentosa With RPGR Gene Variations
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Impaired glutamylation of RPGR ORF15 underlies the cone-dominated phenotype associated with truncating distal ORF15 variants
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Emerging gene therapy products for RPGR-associated X-linked retinitis pigmentosa
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Characterizing the cellular immune response to subretinal AAV gene therapy in the murine retina
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Mirtron-mediated RNA knockdown/replacement therapy for the treatment of dominant retinitis pigmentosa
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Accurate Quantification of AAV Vector Genomes by Quantitative PCR
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Inclusion of PF68 Surfactant Improves Stability of rAAV Titer when Passed through a Surgical Device Used in Retinal Gene Therapy
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CRISPR Interference–Potential Application in Retinal Disease
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Initial results from a first-in-human gene therapy trial on X-linked retinitis pigmentosa caused by mutations in RPGR
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RPGR gene therapy presents challenges in cloning the coding sequence
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Retinal Gene Therapy in X-Linked Retinitis Pigmentosa Caused by Mutations in RPGR: Results at 6 Months in a First in Human Clinical Trial
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Molecular Strategies for RPGR Gene Therapy
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A Quantitative Chloride Channel Conductance Assay for Efficacy Testing of AAV.BEST1
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