Abstract
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.
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Acknowledgments
This work was supported by grants from the Medical Research Council, UK, Fight for Sight, UK, and the Wellcome Trust, UK.
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Orlans, H.O., Edwards, T.L., De Silva, S.R., PatrĂcio, M.I., MacLaren, R.E. (2018). Human Retinal Explant Culture for Ex Vivo Validation of AAV Gene Therapy. In: Boon, C., Wijnholds, J. (eds) Retinal Gene Therapy. Methods in Molecular Biology, vol 1715. Humana Press, New York, NY. https://doi.org/10.1007/978-1-4939-7522-8_21
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DOI: https://doi.org/10.1007/978-1-4939-7522-8_21
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