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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

DOI

10.1007/978-1-4939-7522-8_21

Type

Chapter

Publication Date

2018

Volume

1715

Pages

289 - 303

Keywords

AAV, Culture, Gene therapy, Human retina