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Programmable RNA editing with endogenous ADAR enzymes – a feasible option for the treatment of inherited retinal disease?

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Bellingrath J-S. et al, (2023), Frontiers in Molecular Neuroscience, 16

Impaired glutamylation of RPGR ORF15 underlies the cone-dominated phenotype associated with truncating distal ORF15 variants

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Cehajic-Kapetanovic J. et al, (2022), Proceedings of the National Academy of Sciences, 119

Minicircle Delivery to the Neural Retina as a Gene Therapy Approach.

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Staurenghi F. et al, (2022), Int J Mol Sci, 23

New CRISPR Tools to Correct Pathogenic Mutations in Usher Syndrome.

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Major L. et al, (2022), Int J Mol Sci, 23

CRISPR DNA Base Editing Strategies for Treating Retinitis Pigmentosa Caused by Mutations in Rhodopsin

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Kaukonen M. et al, (2022), Genes, 13, 1327 - 1327

Tropism of AAV Vectors in Photoreceptor-Like Cells of Human iPSC-Derived Retinal Organoids.

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McClements ME. et al, (2022), Transl Vis Sci Technol, 11

Mirtron-mediated RNA knockdown/replacement therapy for the treatment of dominant retinitis pigmentosa

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Orlans HO. et al, (2021), Nature Communications, 12

CRISPR Systems Suitable for Single AAV Vector Delivery.

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Stevanovic M. et al, (2021), Curr Gene Ther

Characterizing the cellular immune response to subretinal AAV gene therapy in the murine retina

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Chandler LC. et al, (2021), Molecular Therapy - Methods & Clinical Development, 22, 52 - 65

Therapy Approaches for Stargardt Disease

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Piotter E. et al, (2021), Biomolecules, 11, 1179 - 1179

Insights on the Regeneration Potential of Müller Glia in the Mammalian Retina

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Salman A. et al, (2021), Cells, 10, 1957 - 1957

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