Recombinant adeno-associated viral vectors as therapeutic agents to treat neurological disorders
Recombinant adeno-associated virus (rAAV) is derived from a small human parvovirus with an excellent safety profile. In addition, this viral vector efficiently transduces and supports long-term transgene expression in the nervous system. These properties make rAAV a reasonable candidate vector for treating neurological disorders. Indeed, rAAV is currently being used in five early stage clinical trials for various neurodegenerative disorders. Therefore, we will review the currently available preclinical data using rAAV in animal models of central nervous system (CNS) disorders. Moreover, potential caveats for rAAV-based gene therapy in the CNS are also presented. Copyright © The American Society of Gene Therapy.
Alzheimer disease, Amyotrophic lateral sclerosis, Epilepsy, Huntington disease, Lysosomal storage disorder, Parkinson disease, Spinal cord, Stroke
Digital Object Identifier (DOI)
Mandel, Ronald J.; Manfredsson, Fredric P.; Foust, Kevin D.; Rising, Aaron; Reimsnider, Sharon; Nash, Kevin; and Burger, Corinna, "Recombinant adeno-associated viral vectors as therapeutic agents to treat neurological disorders" (2006). Translational Neuroscience. 1446.