title: Adeno-associated viral vectors for gene therapy of inherited retinal degenerations.
abstract: Adeno-associated virus (AAV) vectors are in wide use for in vivo gene transfer for the treatment of inherited retinal disease. AAV vectors have been tested in many animal models and have demonstrated efficacy with low toxicity. In this chapter we describe some of the recent methods for small-scale production of these vectors for use in a laboratory setting in volumes and purity appropriate for testing in small and large animals.
authors.affiliation: Neuroscience Division, Department of Molecular and Cell Biology, Helen Wills Neuroscience Institute, University of California, Berkeley, CA, USA. flannery@berkeley.edu
authors.forename: John G
authors.initials: JG
authors.lastname: Flannery
authors.forename: Meike
authors.initials: M
authors.lastname: Visel
mesh_terms: D000818:Animals; D000229:Dependovirus / Q000235:genetics*; D018014:Gene Transfer Techniques; D015316:Genetic Therapy / Q000379:methods; D005822:Genetic Vectors / Q000008:administration & dosage* / Q000235:genetics; D006801:Humans; D012162:Retinal Degeneration / Q000235:genetics* / Q000628:therapy