title: Genetic therapy for the nervous system.
abstract: Genetic therapy is undergoing a renaissance with expansion of viral and synthetic vectors, use of oligonucleotides (RNA and DNA) and sequence-targeted regulatory molecules, as well as genetically modified cells, including induced pluripotent stem cells from the patients themselves. Several clinical trials for neurologic syndromes appear quite promising. This review covers genetic strategies to ameliorate neurologic syndromes of different etiologies, including lysosomal storage diseases, Alzheimer's disease and other amyloidopathies, Parkinson's disease, spinal muscular atrophy, amyotrophic lateral sclerosis and brain tumors. This field has been propelled by genetic technologies, including identifying disease genes and disruptive mutations, design of genomic interacting elements to regulate transcription and splicing of specific precursor mRNAs and use of novel non-coding regulatory RNAs. These versatile new tools for manipulation of genetic elements provide the ability to tailor the mode of genetic intervention to specific aspects of a disease state.
authors.affiliation: Department of Neurology, Center for Neural Development and Disease, University of Rochester, School of Medicine and Dentistry, Rochester, NY 14642, USA.
authors.forename: William J
authors.initials: WJ
authors.lastname: Bowers
authors.forename: Xandra O
authors.initials: XO
authors.lastname: Breakefield
authors.forename: Miguel
authors.initials: M
authors.lastname: Sena-Esteves
mesh_terms: D000544:Alzheimer Disease / Q000628:therapy; D015316:Genetic Therapy / Q000379:methods*; D005822:Genetic Vectors / Q000235:genetics; D006801:Humans; D057026:Induced Pluripotent Stem Cells / Q000166:cytology / Q000378:metabolism; D016464:Lysosomal Storage Diseases / Q000628:therapy; D009422:Nervous System Diseases / Q000628:therapy*; D019636:Neurodegenerative Diseases / Q000628:therapy*; D010300:Parkinson Disease / Q000628:therapy; D012326:RNA Splicing