Ontology highlight
ABSTRACT:
SUBMITTER: Petrs-Silva H
PROVIDER: S-EPMC3034844 | biostudies-other | 2011 Feb
REPOSITORIES: biostudies-other
Molecular therapy : the journal of the American Society of Gene Therapy 20101102 2
Vectors based on adeno-associated virus serotype 2 (AAV2) have been used extensively in many gene-delivery applications, including several successful clinical trials for one type of Leber congenital amaurosis in the retina. Many studies have focused on improving AAV2 transduction efficiency and cellular specificity by genetically engineering its capsid. We have previously shown that vectors-containing single-point mutations of capsid surface tyrosines in serotypes AAV2, AAV8, and AAV9 displayed ...[more]