Engineering Virus-Like Nanoparticles for Targeting the Central Nervous System
University of Sheffield, UK
Keywords: polymersomes, blood brain barrier, axonal transport, intracellular delivery, gene therapy
Abstract:The delivery of therapeutic agents to the central nervous system (CNS) remains a major challenge. Despite the huge effort made to develop novel strategies to overcome the technical challenges. This problem must be overcome to develop fully effective treatments for many conditions, varying from dementia to motor disorders. We have been using use state-of-the-art polymer nanotechnology to engineer biocompatible and nanometer-sized vectors that are able to delivery different cargo within CNS cells without adverse effects. We have shown that these can be used for the delivery of DNA, RNA, antibodys and different drugs. In order to access the CNS we have used two different strategies, one through the blood brain barrier and one exploiting retro-axonal transportation at the muscle-nerve junction. We combined recent advances in both polymer nanotechnology and neuroscience implementing experimental design and biological evaluation with whole body imaging techniques and modelling approaches.