title: Crossing the blood-brain-barrier with viral vectors.
abstract: The abundant vasculature of the CNS provides a compelling route of administration for the delivery of gene therapy vectors if the limitations imposed by the blood-brain-barrier (BBB) can be overcome. There are two general approaches to transporting viral vectors across the BBB: either by transient disruption of brain microvasculature endothelial tight junctions, or through the use of receptor-mediated transcytosis. Advances in BBB disruption have led to pre-clinical success for both global and localized gene delivery, while therapies based on receptor-mediated transcytosis have recently advanced to phase I clinical trials in humans. Both approaches show long term promise for treating a wide range of CNS diseases.
authors: The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA.
authors: Haiyan
authors: H
authors: Fu
authors: The Research Institute at Nationwide Children's Hospital, Columbus, OH, USA; Department of Pediatrics, The Ohio State University College of Medicine, Columbus, OH, USA. Electronic address: douglas.mccarty@nationwidechildrens.org.
authors: Douglas M
authors: DM
authors: McCarty
mesh_terms: D001812:Blood-Brain Barrier; D002493:Central Nervous System Diseases / Q000628:therapy*; D002986:Clinical Trials as Topic; D004337:Drug Carriers / Q000493:pharmacokinetics*; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000493:pharmacokinetics*; D006801:Humans; D010539:Permeability / Q000187:drug effects; D057900:Transcytosis; D014161:Transduction, Genetic; D014780:Viruses / Q000235:genetics* / Q000378:metabolism*
chemical_list: D004337:Drug Carriers