Antibody neutralization poses a barrier to intravitreal adeno-associated viral vector gene delivery to non-human primates.

ABSTRACT: Gene delivery vectors based on adeno-associated viruses (AAV) have exhibited promise in both preclinical disease models and human clinical trials for numerous disease targets, including the retinal degenerative disorders Leber's congenital amaurosis and choroideremia. One general challenge for AAV is that preexisting immunity, as well as subsequent development of immunity following vector administration, can severely inhibit systemic AAV vector gene delivery. However, the role of neutralizing antibodies (NABs) in AAV transduction of tissues considered to be immune privileged, such as the eye, is unclear in large animals. Intravitreal AAV administration allows for broad retinal delivery, but is more susceptible to interactions with the immune system than subretinal administration. To assess

SUBMITTER: Kotterman MA 

PROVIDER: S-EPMC4393652 | biostudies-literature | 2015 Feb

REPOSITORIES: biostudies-literature