The ability to efficiently deliver complete genomic DNA loci to mammalian cells will

have important applications in functional genomics and gene therapy. Recent studies in

our laboratories have led to the development of a novel gene delivery system named the

infectious BAC, or iBAC system, in which human genetic loci cloned as bacterial artificial

chromosomes (BACs), are packaged into herpes simplex virus type 1 (HSV-1) amplicons,

allowing infectious delivery of genomic DNA to a broad range of cell types and tissues

(1-3). Genomic transgenes delivered by iBACs behave in a similar fashion to genes in

their native chromosomal state in terms of expression levels, RNA splicing and promoter

regulation. Because of the natural tropism of HSV-1 for neuronal cells, the iBAC system

may find uses in the treatment of certain neurological disorders. This chapter will discuss

the development of the iBAC system, and its potential application in the treatment of

diseases affecting the central nervous system (CNS).