Adenovirus vectors for human gene therapy

Abstract

Publisher Summary This chapter discusses the structure and biology of adenovirus (Ad) and its exploitation to create a high-level expression vector with use in human gene therapy as one of its most exciting applications. Some of the problems associated with Ad gene therapy, including the host-immune response to Ad, and some potential solutions to these problems are also discussed. The ability of Ad to transduce a wide variety of cell types with high efficiency and the relative ease with which the viral genome can be manipulated genetically make it an ideal vehicle for gene therapy. One of the most promising advancements in Ad gene therapy is the development of helper-dependent Ad vectors, which have maximal cloning capacity, because all of the viral genes have been removed and would be expected to induce a minimal host-immune response. Other recent innovations in vector design described in the chapter include the generation of hybrid Ad vectors to facilitate the transgene integration and manipulations of Ad vectors for targeting specific cell types. First-generation vectors are in many cases, the most effective means at hand for in vivo delivery of foreign genes, and they have proven useful as research tools for efficient gene delivery and expression in vitro .