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Recombinant adeno-associated viral (AAV) vectors have been successfully employed as the mode of gene delivery in several clinical trials for the treatment of inherited retinal diseases to date. The design of such vectors is critical in determining cellular tropism and level of subsequent gene expression that may be achieved following viral delivery. Here we describe a system for living retinal tissue extraction, ex vivo culture, viral transduction and assessment of transgene expression that may be used to assess viral constructs for gene therapy in the human retina at a preclinical stage.

Original publication





Publication Date





289 - 303


AAV, Culture, Gene therapy, Human retina, Dependovirus, Gene Transfer Techniques, Genetic Therapy, Genetic Vectors, Humans, Organ Culture Techniques, Retina, Transduction, Genetic, Transgenes