Ontology highlight
ABSTRACT:
SUBMITTER: Landegger LD
PROVIDER: S-EPMC5340646 | biostudies-literature | 2017 Mar
REPOSITORIES: biostudies-literature
Landegger Lukas D LD Pan Bifeng B Askew Charles C Wassmer Sarah J SJ Gluck Sarah D SD Galvin Alice A Taylor Ruth R Forge Andrew A Stankovic Konstantina M KM Holt Jeffrey R JR Vandenberghe Luk H LH
Nature biotechnology 20170206 3
Efforts to develop gene therapies for hearing loss have been hampered by the lack of safe, efficient, and clinically relevant delivery modalities. Here we demonstrate the safety and efficiency of Anc80L65, a rationally designed synthetic vector, for transgene delivery to the mouse cochlea. Ex vivo transduction of mouse organotypic explants identified Anc80L65 from a set of other adeno-associated virus (AAV) vectors as a potent vector for the cochlear cell targets. Round window membrane injection ...[more]