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As gene therapy of choroideremia is becoming a clinical reality, there is a need for reliable and sensitive assays to determine the expression of exogenously delivered Rab Escort Protein-1 (REP1), in particular to test new gene therapy vectors and as a quality control screen for clinical vector stocks. Here we describe an in vitro protocol to test transgene expression following AAV2/2-REP1 transduction of a human cell line. Gene augmentation can be confirmed by western blot and quantification of the fold-increase of human REP1 levels over untransduced controls.

Original publication

DOI

10.1007/978-1-4939-7522-8_7

Type

Chapter

Publication Date

2018

Volume

1715

Pages

89 - 97

Keywords

AAV, Choroideremia, Gene augmentation, Gene therapy, Human REP1, Western blot, Adaptor Proteins, Signal Transducing, Choroideremia, Dependovirus, Gene Transfer Techniques, Genetic Therapy, Genetic Vectors, HEK293 Cells, Humans, Retina