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Gene Therapy

Study results from University of Minnesota, Medical Department in the area of gene therapy published

Published in Gene Therapy Weekly, September 25th, 2008

"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'')," scientists in the United States report.

"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...

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