Recombinant adenovirus: a gene transfer vector for study and treatment of CNS diseases.

Gene transfer to the CNS with recombinant adenoviral vectors is a relatively recent event. In initial reports it was clearly demonstrated that adenoviral vectors can transfer genetic material to multiple cell types within the CNS. The relative ease in generating recombinant adenovirus (Ad) led to feasibility studies in the CNS with application to animal models of inherited disease, neurodegenerative diseases (e.g., Parkinson's and amyotrophic lateral sclerosis), and cerebrovascular disease. In combination with Ad gene transfer to peripheral tissues, these experiments have identified specific limitations and directed further research to improve vector design, formulation, and delivery.