Precise hit: adeno-associated virus in gene targeting.
Vectors based on the adeno-associated virus (AAV) have attracted much attention as potent gene-delivery vehicles, mainly because of the persistence of this non-pathogenic virus in the host cell and its sustainable therapeutic gene expression. However, virus infection can be accompanied by potentially mutagenic random vector integration into the genome. A novel approach to AAV-mediated gene therapy based on gene targeting through homologous recombination allows efficient, high-fidelity, non-mutagenic gene repair in a host cell.
Vasileva
Ana
A
Department of Gene and Cell Medicine, Mount Sinai School of Medicine, New York, USA.
Jessberger
Rolf
R
D000818:Animals; D004260:DNA Repair; D000229:Dependovirus / Q000235:genetics*; D005057:Eukaryotic Cells; D018390:Gene Targeting; D015316:Genetic Therapy; D005822:Genetic Vectors; D011995:Recombination, Genetic; D014161:Transduction, Genetic