title: MR-guided parenchymal delivery of adeno-associated viral vector serotype 5 in non-human primate brain.
abstract: The present study was designed to characterize transduction of non-human primate brain and spinal cord with AAV5 viral vector after parenchymal delivery. AAV5-CAG-GFP (1 × 1013vector genomes per milliliter (vg ml-1)) was bilaterally infused either into putamen, thalamus or with the combination left putamen and right thalamus. Robust expression of GFP was seen throughout infusion sites and also in other distal nuclei. Interestingly, thalamic infusion of AAV5 resulted in the transduction of the entire corticospinal axis, indicating transport of AAV5 over long distances. Regardless of site of injection, AAV5 transduced both neurons and astrocytes equally. Our data demonstrate that AAV5 is a very powerful vector for the central nervous system and has potential for treatment of a wide range of neurological pathologies with cortical, subcortical and/or spinal cord affection.
authors.affiliation: Department of Neurological Surgery, University of California, San Francisco, CA, USA.
authors.forename: L
authors.identifier: 0000-0001-8151-473X
authors.initials: L
authors.lastname: Samaranch
authors.affiliation: Neurobiology, Research and Development, UniQure NV, Amsterdam 1105BA, The Netherlands.
authors.forename: B
authors.initials: B
authors.lastname: Blits
authors.affiliation: Department of Neurological Surgery, University of California, San Francisco, CA, USA.
authors.forename: W
authors.initials: W
authors.lastname: San Sebastian
authors.affiliation: Department of Neurological Surgery, University of California, San Francisco, CA, USA.
authors.forename: P
authors.initials: P
authors.lastname: Hadaczek
authors.affiliation: Department of Neurological Surgery, University of California, San Francisco, CA, USA.
authors.forename: J
authors.initials: J
authors.lastname: Bringas
authors.affiliation: Department of Neurological Surgery, University of California, San Francisco, CA, USA.
authors.forename: V
authors.initials: V
authors.lastname: Sudhakar
authors.affiliation: Department of Neurological Surgery, University of California, San Francisco, CA, USA.
authors.forename: M
authors.initials: M
authors.lastname: Macayan
authors.affiliation: Department of Neurological Surgery, University of California, San Francisco, CA, USA.
authors.forename: P J
authors.initials: PJ
authors.lastname: Pivirotto
authors.affiliation: Neurobiology, Research and Development, UniQure NV, Amsterdam 1105BA, The Netherlands.
authors.forename: H
authors.initials: H
authors.lastname: Petry
authors.affiliation: Department of Neurological Surgery, University of California, San Francisco, CA, USA.
authors.forename: K S
authors.initials: KS
authors.lastname: Bankiewicz
mesh_terms: D000818:Animals; D001921:Brain / Q000187:drug effects; D000229:Dependovirus / Q000235:genetics; D018014:Gene Transfer Techniques; D015316:Genetic Therapy; D005822:Genetic Vectors / Q000235:genetics / Q000627:therapeutic use*; D049452:Green Fluorescent Proteins / Q000627:therapeutic use; D006801:Humans; D009474:Neurons; D011323:Primates / Q000235:genetics*; D011699:Putamen / Q000000981:diagnostic imaging / Q000378:metabolism; D013116:Spinal Cord / Q000000981:diagnostic imaging / Q000378:metabolism
chemical_list: D049452:Green Fluorescent Proteins