Journal
MOLECULAR THERAPY
Volume 13, Issue 3, Pages 463-483Publisher
CELL PRESS
DOI: 10.1016/j.ymthe.2005.11.009
Keywords
Parkinson disease; Alzheimer disease; Huntington disease; lysosomal storage disorder; amyotrophic lateral sclerosis; spinal cord; epilepsy; stroke
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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.
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