Recombinant adeno-associated virus vector design and gene expression in the mammalian brain.

Efficiency and stability of recombinant adeno-associated virus (rAAV)-mediated gene expression within the mammalian brain are determined by several factors. These include the dose of infectious particles, the purity of the vector stock, the serotype of rAAV, the route of administration, and the intrinsic properties, most notably the rAAV receptor density, of the targeted area. Furthermore, the choice of appropriate regulatory elements in rAAV vector design is of fundamental importance to achieve high-level sustained in vivo transcription and translation. This review summarizes the characteristics of various transcriptional and posttranscriptional regulatory elements, and highlights their influence on the expression performance of rAAV vectors in the mammalian brain.