Gene transfer for cerebrovascular disease

Gene transfer is a powerful, evolving technique that uses a biologic vehicle (eg, an engineered adenovirus) to introduce a specific gene of interest (ie, a recombinant gene) into a target tissue. This approach, which has considerable therapeutic potential, underlies the concept of gene therapy. Several studies have characterized the morphologic, biochemical, and functional effects of recombinant gene expression in animal and human cerebral arteries, and support the possibility of gene therapy for cerebrovascular disease. However, for successful integration into future clinical practice, key issues concerning vector safety, delivery methods, and transduction specificity need to be addressed. Alongside completion of the Human Genome Project, transfer of novel genes into the central nervous system is likely to impact greatly on our ability to favorably modify diseased human tissue. Knowledge of the fundamental concepts of cerebrovascular gene transfer is therefore useful to understanding both its molecular basis and potential clinical utility.