High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors.

ABSTRACT: Vectors derived from adeno-associated viruses (AAVs) have become important gene delivery tools for the treatment of many inherited ocular diseases in well-characterized animal models. Previous studies have determined that the viral capsid plays an essential role in the cellular tropism and efficiency of transgene expression. Recently, it was shown that phosphorylation of surface-exposed tyrosine residues from AAV2 capsid targets the viral particles for ubiquitination and proteasome- mediated degradation, and mutations of these tyrosine residues lead to highly efficient vector transduction in vitro and in vivo. Because the tyrosine residues are highly conserved in other AAV serotypes, in this study we evaluated the intraocular transduction characteristics of vectors containing point mutatio

SUBMITTER: Petrs-Silva H 

PROVIDER: S-EPMC2835095 | biostudies-literature | 2009 Mar

REPOSITORIES: biostudies-literature