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Rapid and Efficient Generation of Recombinant Human Pluripotent Stem Cells by Recombinase-mediated Cassette Exchange in the AAVS1 Locus
Published on: November 20, 2016
RNA-polymerase III-driven expression cassettes in human gene therapy
1Department of Medical Genetics and Microbiology, Faculty of Medicine, University of Toronto, Toronto, Ontario M5S 3E2, Canada.
Abstract:
tRNAs, U6 small nuclear RNA and adenovirus virus associated RNAs are transcribed by RNA polymerase III. These RNAs are expressed in high levels and possess increased stability due to their small, compact size. Promoters expressing these RNAs have therefore been used to express a variety of interfering RNAs, including ribozymes, antisense RNAs and sense or decoy RNAs. Intracellular expression and activity of RNA polymerase III-driven interfering RNAs have been demonstrated in several studies. This review will summarize the design and application of RNA polymerase III-driven expression cassettes in human gene therapy using retroviral vectors as the primary method of gene delivery.
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