Recombinant adeno-associated viral vectors in the nervous system.
Recombinant adeno-associated virus 2 (rAAV2) has been extensively used as a gene delivery vector for the nervous system. It targets primarily neurons in the nervous system and results in sustained long-term expression of transgenes. New rAAV serotypes have been characterized and demonstrated to have improved transduction efficiencies in various regions of the brain and spinal cord. This review discusses some properties of rAAV that have been studied in the nervous system such as cell tropism, duration of transgene expression, and distribution of viral transduction, as well as immunity and regulation of transgene expression issues, all of which are important for optimization of the use of rAAV in the nervous system.
Burger
Corinna
C
Department of Molecular Genetics and Microbiology, College of Medicine, University of Florida, Gainesville, FL 32610, USA. cburger@ufl.edu
Nash
Kevin
K
Mandel
Ronald J
RJ
D000818:Animals; D002490:Central Nervous System / Q000378:metabolism* / Q000821:virology; D000229:Dependovirus / Q000235:genetics*; D005786:Gene Expression Regulation; D015316:Genetic Therapy; D005822:Genetic Vectors; D008353:Mannitol / Q000627:therapeutic use; D009422:Nervous System Diseases / Q000628:therapy*; D014161:Transduction, Genetic / Q000379:methods; D019076:Transgenes
D008353:Mannitol