ABSTRACT
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).
