Abstract

This chapter discusses the design of the recombinant adeno-associated virus (rAAV) expression cassette and the selection of the rAAV serotype. The minimum requirements of a rAAV expression cassette are a promoter, a transgene, and a polyadenylation site flanked on either end by AAV inverted terminal repeats. Many promoters have been isolated that differ in cell-type specificity, size, and strength. In addition, novel AAV serotypes are continually being isolated and characterized in vivo, differs in their cell-type specific tropism. The efficiency of cellular transduction and the level and spread of gene expression is mediated by the recombinant vectors. The chapter provides an introduction of the promoters, regulatory elements, and serotypes that are available, and a guide to assist in the design of rAAV cassettes and selection of the appropriate rAAV serotype for a particular application. The criterion for selection of a particular promoter and regulatory element(s) is based on the size of the transgene, the cell type to be transduced, and the level of gene expression required for a particular application.

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