title: Molecular neurosurgery: vectors and vector delivery strategies.
abstract: Molecular neurosurgery involves the use of vector-mediated gene therapy and gene knockdown to manipulate in vivo gene expression for the treatment of neurological diseases. These techniques have the potential to revolutionise the practice of neurosurgery. However, significant challenges remain to be overcome before these techniques enter routine clinical practice. These challenges have been the subject of intensive research in recent years and include the development of strategies to facilitate effective vector delivery to the brain and the development of both viral and non-viral vectors that are capable of efficient cell transduction without excessive toxicity. This review provides an update on the practice of molecular neurosurgery with particular focus on the practical neurosurgical aspects of vector delivery to the brain. In addition, an introduction to the key vectors employed in clinical trials and a brief overview of previous gene therapy clinical trials is provided. Finally, key areas for future research aimed at increasing the likelihood of the successful translation of gene therapy into clinical trials are highlighted.
authors.affiliation: Department of Neurosurgery, Frenchay Hospital, Bristol, UK. ew296@doctors.net.uk
authors.forename: Edward
authors.initials: E
authors.lastname: White
mesh_terms: D001921:Brain / Q000378:metabolism / Q000473:pathology* / Q000601:surgery*; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000008:administration & dosage*; D006801:Humans; D058990:Molecular Targeted Therapy / Q000379:methods* / Q000592:standards; D009493:Neurosurgery / Q000379:methods* / Q000592:standards