Ontology highlight
ABSTRACT:
SUBMITTER: Miyanohara A
PROVIDER: S-EPMC4943453 | biostudies-literature | 2016
REPOSITORIES: biostudies-literature
Miyanohara Atsushi A Kamizato Kota K Juhas Stefan S Juhasova Jana J Navarro Michael M Marsala Silvia S Lukacova Nada N Hruska-Plochan Marian M Curtis Erik E Gabel Brandon B Ciacci Joseph J Ahrens Eric T ET Kaspar Brian K BK Cleveland Don D Marsala Martin M
Molecular therapy. Methods & clinical development 20160713
Effective in vivo use of adeno-associated virus (AAV)-based vectors to achieve gene-specific silencing or upregulation in the central nervous system has been limited by the inability to provide more than limited deep parenchymal expression in adult animals using delivery routes with the most clinical relevance (intravenous or intrathecal). Here, we demonstrate that the spinal pia membrane represents the primary barrier limiting effective AAV9 penetration into the spinal parenchyma after intrathe ...[more]