title: Adeno-associated virus vectors and neurological gene therapy.
abstract: Gene therapy has strong potential for treating a variety of genetic disorders, as demonstrated in recent clinical trials. There is unfortunately no scarcity of disease targets, and the grand challenge in this field has instead been the development of safe and efficient gene delivery platforms. To date, approximately two thirds of the 1800 gene therapy clinical trials completed worldwide have used viral vectors. Among these, adeno-associated virus (AAV) has emerged as particularly promising because of its impressive safety profile and efficiency in transducing a wide range of cell types. Gene delivery to the CNS involves both considerable promise and unique challenges, and better AAV vectors are thus needed to translate CNS gene therapy approaches to the clinic. This review discusses strategies for vector design, potential routes of administration, immune responses, and clinical applications of AAV in the CNS.
authors.affiliation: Department of Chemical and Biomolecular Engineering, University of California, Berkeley, CA, USA.
authors.forename: David S
authors.initials: DS
authors.lastname: Ojala
authors.affiliation: Department of Molecular and Cell Biology, University of California, Berkeley, CA, USA.
authors.forename: Dominic P
authors.initials: DP
authors.lastname: Amara
authors.affiliation: Department of Chemical and Biomolecular Engineering, University of California, Berkeley, CA, USA Department of Bioengineering, University of California, Berkeley, CA, USA The Helen Wills Neuroscience Institute, University of California, Berkeley, CA, USA schaffer@berkeley.edu.
authors.forename: David V
authors.initials: DV
authors.lastname: Schaffer
mesh_terms: D000818:Animals; D002490:Central Nervous System / Q000821:virology*; D002493:Central Nervous System Diseases / Q000628:therapy*; D000229:Dependovirus / Q000235:genetics*; D015316:Genetic Therapy; D005822:Genetic Vectors / Q000008:administration & dosage; D006801:Humans
keywords: adeno-associated virus (AAV); central nervous system; directed evolution; gene therapy; viral vectors