Ontology highlight
ABSTRACT:
SUBMITTER: Wang D
PROVIDER: S-EPMC5948233 | biostudies-literature | 2018 Jun
REPOSITORIES: biostudies-literature
Wang Dan D Li Shaoyong S Gessler Dominic J DJ Xie Jun J Zhong Li L Li Jia J Tran Karen K Van Vliet Kim K Ren Lingzhi L Su Qin Q He Ran R Goetzmann Jason E JE Flotte Terence R TR Agbandje-McKenna Mavis M Gao Guangping G
Molecular therapy. Methods & clinical development 20180316
Adeno-associated virus (AAV) has provided the gene therapy field with the most powerful <i>in vivo</i> gene delivery vector to realize safe, efficacious, and sustainable therapeutic gene expression. Because many clinically relevant properties of AAV-based vectors are governed by the capsid, much research effort has been devoted to the development of AAV capsids for desired features. Here, we combine AAV capsid discovery from nature and rational engineering to report an AAV9 capsid variant, desig ...[more]