Molecular therapeutic strategies in neurodegenerative diseases and injury

Abstract Molecular therapeutics for use in neurodegenerative disease and traumatic injury to the central nervous system have been implemented widely in preclinical studies and clinical trials. These strategies have broadly employed both pharmacologic and vector-driven gene-therapy approaches targeting different pathophysiologic mechanisms. As such, we have selected several representative indications that can serve as examples of traumatic, autoimmune, and mixed pathophysiologic mechanisms. The aim of this chapter is to provide an overview of molecular therapeutic options in preclinical and clinical development in several representative indications of neurodegenerative disease and injury. Here we will discuss molecular therapies in development for amyotrophic lateral sclerosis, multiple sclerosis, Alzheimer’s disease, Parkinson’s disease, traumatic brain injury, and traumatic spinal cord injury.