Ku70, an essential gene, modulates the frequency of rAAV-mediated gene targeting in human somatic cells.

ABSTRACT: Gene targeting has two important applications. One is the inactivation of genes ("knockouts"), and the second is the correction of a mutated allele back to wild-type ("gene therapy"). Central to these processes is the efficient introduction of the targeting DNA into the cells of interest. In humans, this targeting is often accomplished through the use of recombinant adeno-associated virus (rAAV). rAAV is presumed to use a pathway of DNA double-strand break (DSB) repair termed homologous recombination (HR) to mediate correct targeting; however, the specifics of this mechanism remain unknown. In this work, we attempted to generate Ku70-null human somatic cells by using a rAAV-based gene knockout strategy. Ku70 is the heterodimeric partner of Ku86, and together they constitute an end-binding

SUBMITTER: Fattah FJ 

PROVIDER: S-EPMC2438404 | biostudies-literature | 2008 Jun

REPOSITORIES: biostudies-literature