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.
Mandel
Ronald J
RJ
Department of Neuroscience, and McKnight Brain Institute, University of Florida College of Medicine, Gainesville, FL 32610, USA. rmandel@ufl.edu
Manfredsson
Fredric P
FP
Foust
Kevin D
KD
Rising
Aaron
A
Reimsnider
Sharon
S
Nash
Kevin
K
Burger
Corinna
C
D000818:Animals; D004274:DNA, Recombinant / Q000627:therapeutic use*; D000229:Dependovirus / Q000235:genetics*; D015316:Genetic Therapy / Q000379:methods* / Q000639:trends; D005822:Genetic Vectors / Q000627:therapeutic use*; D006801:Humans; D019636:Neurodegenerative Diseases / Q000235:genetics / Q000378:metabolism / Q000628:therapy*
D004274:DNA, Recombinant