Application to Gene Therapy and Vaccination

Central nervous system (CNS) diseases represent a class of complex disorders for which cures have been largely unmet due to the general lack of knowledge regarding underlying pathogenic mechanisms. Gene-based therapies directed at ameliorating neurodegenerative diseases exhibit great potential due to rapid scientific advances made regarding delivery modalities, neurosurgical methods, neuroimaging, and molecular biological manipulation. Given these breakthroughs, the diseased CNS presents a neuroimmunological challenge as gene delivery many times requires invasive surgical procedures and a majority of the gene delivery platforms incite transient, and sometimes, inflammatory events that possess the potential to exacerbate disease-related processes. In this chapter, we will discuss the most current literature on gene therapy for CNS disorders by detailing the neuroimmunological profiles of presently available gene transfer platforms, approaches that have been made to minimize vector-mediated inflammation, and current state of gene therapy clinical trials for neurodegenerative diseases.