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

Journal article

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

Journal article

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

Therapy Approaches for Stargardt Disease

Journal article

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

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

Journal article

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

Accurate Quantification of AAV Vector Genomes by Quantitative PCR

Journal article

Martinez-Fernandez de la Camara C. et al, (2021), Genes, 12, 601 - 601

Genome-Editing Strategies for Treating Human Retinal Degenerations

Journal article

Quinn J. et al, (2021), Human Gene Therapy, 32, 247 - 259

AAV Induced Expression of Human Rod and Cone Opsin in Bipolar Cells of a Mouse Model of Retinal Degeneration

Journal article

McClements ME. et al, (2021), BioMed Research International, 2021, 1 - 8

Is subretinal AAV gene replacement still the only viable treatment option for choroideremia?

Journal article

Han RC. et al, (2021), Expert Opinion on Orphan Drugs, 9, 13 - 24

An analysis of the Kozak consensus in retinal genes and its relevance to gene therapy.

Journal article

McClements ME. et al, (2021), Mol Vis, 27, 233 - 242

CRISPR genome engineering for retinal diseases

Chapter

Kantor A. et al, (2021), Progress in Molecular Biology and Translational Science

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