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Gene Therapy Weekly

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Artificial Chromosomes



Studies from Royal Children's Hospital provide new data on artificial chromosomes



December 18th, 2008

According to recent research from Parkville, Australia, "The stable, regulated and tissue-specific expression of a therapeutic transgene can be best achieved by the transfer of a complete genomic locus, which will include the short-and long-range regulatory elements that are critical for the accurate control of gene expression. However, when techniques that rely on the random integration of exogenous DNA into the human genome are used for gene transfer, the risk of insertional mutagenesis remains a major issue."

"Using components derived from the adeno-associated virus (AAV), we have successfully targeted the integration of 200 kb bacterial artificial chromosomes...


Source: Gene Therapy Weekly (2008-12-18)

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