Neurosurgical Targeting, Delivery, and Infusion of Gene Therapy Agents in the Brain

This chapter reviews the gene therapy in the brain for different vectors, innumerable targets, and a plethora of neurological diseases. Human gene transfer to the brain depends on the methodology of delivery and the mechanics of infusion. The method of delivery may differ according to the surgical goal. Anatomical targeting represents the tools, the reliability, and preciseness of the method to facilitate accurate surgical planning. Current methodology limits the clinical utility of delivering gene therapy from a peripheral administration, so a proper targeting of an infusion catheter is necessary so that the intended target cell population is treated. The variables that alter infusion modify the efficacy of the final product. Gene therapy continues to move into clinical practice, continued evolution of surgical techniques and infusion devices will aid in the safe and effective translation of biologically promising agents. The experiences with human trials in which a small focal area of the brain is targeted, such as Parkinson's disease, has revealed very different surgical delivery requirements compared with diseases, where global delivery may be desirable, such as the genetic disorder of Batten disease.